GSK announced it will begin shipping its quadrivalent influenza vaccines to US healthcare providers and pharmacies for the 2018-19 flu season, immediately following licensing and lot-release approval from the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research.
“The 2017-18 flu season was an important reminder that the flu is a serious and unpredictable disease,” said Dr. Leonard Friedland, VP, director of Scientific Affairs and Public Health, Vaccines, North America. “Annual vaccination can help prevent flu illnesses and flu-related hospitalizations and reduce the severity of the disease. The more people of all ages who are immunized, the less chance the virus has to spread and the better we can help protect against the flu this season.”
The US Centers for Disease Control and Prevention (CDC) estimates that flu vaccination during the 2016-17 flu season prevented an estimated 5.29 million illnesses, 2.64 million medical visits and 84,700 hospitalizations associated with flu, underscoring the benefits of current vaccines. Yet, according to the CDC, approximately 80 per cent of flu-related deaths in children during the 2017-18 season occurred in children who were not vaccinated against the flu that season.
“The flu vaccine is the one vaccine that people of almost all ages should receive annually,” said Patrick Desbiens, senior vice president, US Vaccines. “GSK is committed to helping healthcare professionals provide flu vaccinations across the lifespan. Beginning with the 2018-19 flu season, we can now offer two vaccines that enable providers to vaccinate all of their recommended patients aged 6 months and older with the same vaccine dose."
GSK expects to supply up to 40 to 45 million total doses across both of its vaccines for the US market in the 2018-19 season, the highest volume of doses planned for distribution by GSK to date. FLULAVAL QUADRIVALENT will be available in a 5mL, multidose vial containing 10 doses (0.5mL each) and a 0.5mL, single-dose, prefilled syringe. FLUARIXQUADRIVALENT will be available in a 0.5mL, single-dose, prefilled syringe.
For the 2018-19 flu season, the World Health Organization (WHO) and FDA’s Vaccines and Related Biological Products Advisory Committee recommended including the A/Michigan/45/2015 (H1N1) pdm09-like virus, A/Singapore/INFIMH-16-0019/2016 (H3N2)-like virus and B/Colorado/06/2017-like virus, with the addition of B/Phuket/3073/2013-like virus for the quadrivalent vaccine.
About seasonal influenza
Seasonal influenza is a contagious respiratory illness, caused by flu viruses. There are two main types of flu viruses, A and B, that spread between people and can cause mild to severe illness. Most flu activity in the US occurs from October through May, and it usually peaks between December and February.
While anyone can get the flu, it can be particularly serious for young children, older people, pregnant women and people with certain health conditions, such as asthma. According to the CDC, the best tool available to help protect yourself and those around you against the flu is to get vaccinated. The more people who are vaccinated, the less chance the virus has to spread. The CDC recommends that all people over the age of 6 months get vaccinated against the flu annually.
Perrigo Company plc has entered into a licensing agreement with a subsidiary of Merck & Co, Inc., Kenilworth, NJ, USA for the exclusive rights in the United States to pursue regulatory approval for a non-prescription, over-the-counter (OTC) Nasonex (mometasone furoate monohydrate) nasal spray.
Under the terms of the agreement, Perrigo holds exclusive rights to market, sell, and distribute a non-prescription version of Nasonex OTC in the United States following Perrigo's receipt of all necessary regulatory approvals. Financial terms of the licensing agreement were not disclosed.
Nasonex is currently available by prescription only. Annual prescription brand and generic market sales for the 12 months ending June 2018 were approximately USD 214 million as measured by IQVIA.
Perrigo president and CEO Uwe Roehrhoff commented, "We are excited to enter into this agreement with Merck & Co, Inc., Kenilworth, NJ, USA. This announcement exemplifies inorganic opportunities to expand the OTC self-care market while enhancing long-term growth in our US consumer business. Leveraging our innovative product development capabilities and efficient supply chain enables us to enhance our durable OTC portfolio by establishing a framework to actively participate in future switch opportunities. Perrigo is well positioned to provide quality affordable healthcare solutions to patients and families."
Perrigo sxecutive vice president and president, Consumer Healthcare Americas Jeff Needham stated, "This product in-license is the first of its kind for Perrigo. As other similar products that have previously switched from prescription to OTC status, we are working diligently to bring this important product to consumers and customers more quickly than the average 5-year OTC switch timeframe. This strategic investment into the OTC category creates an innovative product offering for Perrigo. We expect to execute the Rx-OTC-switch, fully penetrate this market with a branded offering and provide a future store brand alternative."
Perrigo Company plc, a leading global healthcare company, delivers value to its customers and consumers by providing Quality Affordable Healthcare Products. Founded in 1887 as a packager of home remedies, Perrigo has built a unique business model that is best described as the convergence of a fast-moving consumer goods company, a high-quality pharmaceutical manufacturing organization and a world-class supply chain network. Perrigo is one of the world's largest manufacturers of over-the-counter (OTC) healthcare products and suppliers of infant formulas for the store brand market. The Company also is a leading provider of branded OTC products throughout Europe and the US, as well as a leading producer of "extended topical" prescription drugs. Perrigo, headquartered in Ireland, sells its products primarily in North America and Europe, as well as in other markets, including Australia, Israel and China.
The US Food and Drug Administration (FDA) has approved Poteligeo (mogamulizumab-kpkc) injection for intravenous use for the treatment of adult patients with relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS) after at least one prior systemic therapy. This approval provides a new treatment option for patients with MF and is the first FDA approval of a drug specifically for SS.
“Mycosis fungoides and Sézary syndrome are rare, hard-to-treat types of non-Hodgkin lymphoma and this approval fills an unmet medical need for these patients,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “We are committed to continuing to expedite the development and review of this type of targeted therapy that offers meaningful treatments for patients.”
Non-Hodgkin lymphoma is a cancer that starts in white blood cells called lymphocytes, which are part of the body's immune system. MF and SS are types of non-Hodgkin lymphoma in which lymphocytes become cancerous and affect the skin. MF accounts for about half of all lymphomas arising from the skin. It causes itchy red rashes and skin lesions and can spread to other parts of the body. SS is a rare form of skin lymphoma that affects the blood and lymph nodes.
Poteligeo is a monoclonal antibody that binds to a protein (called CC chemokine receptor type 4 or CCR4) found on some cancer cells.
The approval was based on a clinical trial of 372 patients with relapsed MF or SS who received either Poteligeo or a type of chemotherapy called vorinostat. Progression-free survival (the amount of time a patient stays alive without the cancer growing) was longer for patients taking Poteligeo (median 7.6 months) compared to patients taking vorinostat (median 3.1 months).
The most common side effects of treatment with Poteligeo included rash, infusion-related reactions, fatigue, diarrhea, musculoskeletal pain and upper respiratory tract infection.
Serious warnings of treatment with Poteligeo include the risk of dermatologic toxicity, infusion reactions, infections, autoimmune problems (a condition where the immune cells in the body attack other cells or organs in the body), and complications of stem cell transplantation that uses donor stem cells (allogeneic) after treatment with the drug.
The FDA granted this application priority review and breakthrough therapy designation. Poteligeo also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
The FDA granted this approval to Kyowa Kirin, Inc.
The FDA, an agency within the US Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
United Therapeutics Corporation has entered into a Settlement Agreement with Watson Laboratories, Inc. (Watson) resolving ongoing litigation concerning certain patents relating to United Therapeutics' product, Tyvaso (treprostinil) Inhalation Solution, and Watson's Abbreviated New Drug Application (ANDA) seeking approval by the US Food and Drug Administration to market a generic version of Tyvaso.
Under the Settlement Agreement, United Therapeutics granted to Watson a license under its patent rights to manufacture and commercialize the generic version of Tyvaso described in its ANDA filing in the United States beginning on January 1, 2026, although Watson may be permitted to enter the market earlier under certain circumstances.
The license included in the Settlement Agreement does not permit Watson to manufacture a generic version of any other United Therapeutics product, such as Remodulin (treprostinil) Injection or Orenitram (treprostinil) Extended-Release Tablets. The Settlement Agreement does not grant Watson any rights other than those required to launch Watson's generic version of Tyvaso.
In accordance with the Settlement Agreement, the parties will submit the Settlement Agreement to the US Federal Trade Commission and the US Department of Justice for review and will also terminate the pending litigation concerning patents relating to Watson's ANDA.
United Therapeutics Corporation is a biotechnology company focused on the development and commercialization of innovative products to address the unmet medical needs of patients with chronic and life-threatening conditions.
Teva Pharmaceutical Industries Ltd., announced the launch of a generic version of Uceris (budesonide) extended-release tablets, 9 mg, in the US.
Budesonide extended-release tablets are a glucocorticosteroid indicated for the induction of remission in patients with active, mild to moderate ulcerative colitis.
“The launch of generic budesonide extended-release tablets signals an important addition to Teva’s portfolio,” said Brendan O’Grady, EVP and head of North America Commercial. “We continue to be focused on bringing affordable generic treatment options to our customers, including those living with chronic, life-long conditions like ulcerative colitis.”
With nearly 550 generic medicines available, Teva has the largest portfolio of FDA-approved generic products on the market and holds the leading position in first-to-file opportunities, with over 100 pending first-to-files in the US. Currently, one in seven generic prescriptions dispensed in the US is filled with a Teva generic product.
Uceris had annual sales of approximately $196 million in the US, according to IMS data as of May 2018.
The Korean pharma market is experiencing significant growth in finished drug products, according to CPhI Korea. Over the last 12 months, market conditions have spurred a strong expansion in exports of finished formulations, driven primarily by an expanding domestic generics market.
South Korea’s domestic companies are transitioning quickly to an export-oriented stance. According to the Korean Pharmaceutical Traders Association (KPTA), the export of domestic finished products is growing with a total volume of $2.6 billion in 2017. Domestic APIs are also seeing steady growth with a total volume of $1.6 billion exported in 2017.
“The rapid internationalisation of Korea has occurred as a result of the competitive pricing in the large domestic generics market, which has made Korean exports increasing attractive. What we are now seeing is Korean companies targeting regional exports and even opening facilities overseas to capitalise on growth. As a result, we have seen a steady increase in international attendees and finished formulation companies” commented Laura Murina, CPhI Korea Brand Manager.
CPhI Korea – organised by UBM (part of Informa PLC) – will host its fifth annual event at the Seoul COEX Convention and Exhibition Centre from August 28-30, 2018. The show is a bellwether of wider trends in the country and this year is set to launch finished dosage formulation (FDF) zone in response to the growing demand. Over 5000 executives from 50 countries and more than 175 exhibitors will attend what is becoming a regional distribution hub for Asian pharmaceuticals.
Additionally, in the last few years, co-located BioPh has become a focus of global and domestic attendee interest. South Korea is now a hot bed of biosimilar development and is on the cutting-edge of innovation with multiple European approvals.
Enhanced onsite features and new conference programmes at CPhI Korea 2018 will be dedicated to helping attendees navigate the Korean market and changing trends. The agenda explores the ‘implementation of ICH Q3D for APIs’, ‘USA complex generic & biosimilar regulatory approval process’, and emphasizing the internationalization of the market, a session from the PMDA on ‘future perspectives of GMP in Japan’.
With many international companies also looking to register products in Korea, the event has partnered with the KPTA for ‘meet the expert’ sessions to facilitate increased international-domestic partnerships.
Murina added, “South Korea has an expanding reputation for delivering high quality pharma, and global companies are now taking advantage of quicker clinical trials, the niche biosimilar industry, and innovative local companies to develop partnerships. As a result, the API sector will continue to grow steadily, with domestic companies increasing exports. However, we are simultaneously seeing international companies take advantage of changing regulations and demographics to enter the market.”
Pune-based healthcare startup SynThera Biomedical Private Limited, which focuses on R&D, manufacture and commercialization of affordable biomaterials-based medical devices, has secured an undisclosed amount of seed funding investment from two investors – equity crowd-funding platform 1Crowd and the government’s Biotechnology Industry Research Assistance Council (BIRAC) SEED scheme via Venture Center.
The investment will help us achieve expansion, approvals and launch of our first line of bone graft substitute products in the market, together with further development and testing on our patented bone graft product line, Nilay Lakhkar, founder and chief executive of SynThera Biomedical, said.
The company’s flagship products - SynOst and PoroSyn are synthetic bone graft substitute products made from bioactive glass materials. Both products are used in dental and orthopaedic surgeries as implantable materials for repair and regeneration of bone that is lost or damaged due to disease, injuries or congenital deformities.
“SynThera is an innovative biomaterial sciences company, with patented technology, which we believe can address a global opportunity through an array of technology lead products. 1Crowd seeks to invest in ventures that are at the cusp of knowledge and technology, and SynThera is a fine example of that. With investments in over 17 startups, 1Crowd has built an ecosystem, that startups such as SynThera can leverage, which includes an investor community, a mentorship panel and industry connects,” said Anil Gudibande, co-founder of 1Crowd.
“BIRAC’s SEED – Sustainable Entrepreneurship and Enterprise Development – scheme was set up to support life sciences start-ups, with a funding of up to Rs. 30 lakh. The investments raised by Venture Center's incubatee company SynThera will enable it to move faster towards regulatory approvals, production and sales. SynThera has unique technology capabilities and intellectual property, and will be the first bioactive glass company from India. It will be uniquely positioned to provide valuable biomaterial solutions to dental and orthopaedic surgeons", said Dr. Premnath, director, Venture Center, the incubator that supports SynThera Biomedical.
Venture Center is India’s leading inventive enterprises incubator. It won the national award for technology business incubators from the President of India in May 2016. The Venture Center is hosted by CSIR-National Chemical Laboratory, Pune.
Safe Point India, HMD Foundation hails Punjab CM’s decision to revoke ban on sale of syringes without prescriptionRead Now
Hailing Punjab chief minister Amarinder Singh’s prompt decision to revoke the ban on the sale of syringes without a prescription, Safe Point India and HMD Foundation appreciated and thanked him for the much needed timely action.
After the order Safe Point India and HMD Foundation raised an alarming concern on the urgent need to amplify this order because restrictive practices can actually compel a user to reuse the old easily available syringes and thereby expose himself and all other members of community to various infections and health hazard of undetermined proportions adding to already huge burden of HIV/ hepatitis B /hepatitis C infections and a host of other diseases.
Singh on Monday revoked the ban on the sale of syringes without a prescription. The chief minister’s office has directed deputy commissioners across the state to issue orders to chemists to prepare an inventory of syringes and keep a record of their sale and the customers sold to.
“We do not know why the respective deputy commissioners issued such ban orders. There was no direction from the state government. We will amend it,” said a functionary.
At least three district administrations, Faridkot, Bathinda and Fazilka have issued directives to medical stores to not sell syringes without a doctor’s written prescription. The order issued by the deputy commissioner using the power under section 144 of CrPC will come into effect immediately and last till September 30, 2018.
“Instead of restriction to syringes access it would be more prudent to accelerate a switch to auto disable syringes in Punjab and have a state policy similar to the one by government of Andhra Pradesh to prevent reuse with the help of State Drug Controller to address this magnifying problem” said Pardeep Sareen, Chief general manager-marketing, Hindustan Syringe and Medical Devices Ltd.
Sareen said Punjab has the highest incidence of hepatitis B and C spreading from drug addicts to other patients in the already-challenged healthcare infrastructure.
“The order was distraught with trouble. We are thankful to Singh for timely intervention. While the law exists in the US of syringe sale limited to prescription, this is not the case in India and the intent of solving a problem may be noble but the management tool being used is questionable and the wrong prescription as it will lead to bigger problem of infections being spread from Syringes reused. In other countries they promote free Needle exchange programs to addicts to motivate them not to share Syringes or reuse them,” said Rajiv Nath joint managing director of Hindustan Syringes & Medical Devices.
The disposable syringes available in market are subject to re-use. Numerous and periodic media reports and case studies have identified reuse of syringes and needles as a major cause of spread of diseases.
“In order to prevent reuse and contain spread of infection, it is advisable and recommended to use AD syringes specifically designed to prevent reuse. This reuse prevention has been strongly advocated by WHO, MoH&FW has been seeking manufacturers to add capacity of AD Syringes and the recently concluded HTA study from PGI chandigarh on safety engineered devices under aegis of Punjab government and DHR, MoH, government of India had again proven the cost effectiveness of this intervention ” emphasised Dr Ratti , healthcare advisor, Safe Point India.
“Andhra Pradesh government has already taken a lead in this regard and issued instructions for 100% use of AD syringes in its public health facilities from 28th Jul 2018which is observed as World Hepatitis Day. Currently AD syringes are used in immunization programs only in most states though many central government hospitals in Delhi are using these for years,” Dr. Ratti added.
Safe point India and HMD Foundation which work in areas of promoting safe injection practices through policy advocacy and safe and proper use of injection practices including its safe disposal has been urging central and state governments to adopt use of AD syringes in therapeutic practices also.
Safe point India therefore urged that Punjab government order be suitably amended to include following: All prescriptions must specify use of AD syringes. It would be more prudent to accelerate a switch to auto disable syringes in private sector in Punjab and have a state policy as done by government of Andhra Pradesh (issued instructions for 100% use of AD syringes in its public health facilities from July 28, 2018 which is observed as World Hepatitis Day) to prevent reuse with help of state drugs controller to address this magnifying problem in Punjab.
After holding a review meeting on drugs today, the chief minister will issue detailed guidelines on the sale of syringes to all deputy commissioners.
Eupheus Learning, a Delhi based EdTech start-up and the Department of Mental Health and Behavioural Sciences, Fortis Healthcare, have joined hands to spread awareness about the human psychology issue.
Dr. Samir Parikh, director, Department of Mental Health and Behavioral sciences, Fortis Healthcare, conceptualized PsychEd2018 India’s only National Psychology Quiz for school students. This quiz aims to cultivate an interest in young minds for the field of psychology and promote awareness around mental health issues nationally. Last year, the event had 460 schools from 90 cities across India. Here 1380 students participated for the online round alone.
Being knowledge partners for the contest, Eupheus Learning will be running pre-event contests on their Facebook page to promote the quiz. Together, they will also hold Facebook live sessions of 30 minute each where mental health experts from Fortis Healthcare will elaborately cover topics related to education and mental health.
“Given the recent incidents being highlighted in the media, regarding mental health concerns; it has become our endeavour to reach out to the young population of the country and help them know about psychology and take care of their mental health. Psych-Ed is an engaging platform along with Eupheus Learning which is connected around 1700 schools and enables us to engage an even larger number students this year, said Dr. Parikh.
“We are actively seeking association with events that give students a novel opportunity of enhancing their understanding beyond textbooks. Our association with Fortis Healthcare syncs well with what we are trying to achieve for the future. The next generation of young minds needs to be made aware as well as given support regarding mental health issues that are rampant, said Sarvesh Shrivastava, co-founder and MD, Eupheus Learning.
Euphues Learning caters to pre-K to Class X of 22,000 private schools across India with a base of approximately 26 million students. While the first live Facebook interaction will take place on the July 20,, 2018 with Kamna Chhibber. The second live Facebook interaction scheduled for August 10, 2018 with Dr. Parikh.
Axovant Sciences announced that it has licensed exclusive global rights to an investigational Silence-and-Replace gene therapy program from Benitec Biopharma for the treatment of oculopharyngeal muscular dystrophy (OPMD), and has also entered into a research collaboration for the development of five additional gene therapy products in neurological disorders.
The Silence-and-Replace gene therapy technology is designed to deliver a combination of DNA-directed RNA interference (silence) along with a functional copy of the gene (replace) in a single vector construct. This approach is applicable to various genetic diseases, including autosomal dominant disorders caused by nucleotide repeat expansion.
The lead program, AXO-AAV-OPMD, is in preclinical development, and Axovant plans to initiate a placebo-controlled clinical study in 2019. OPMD is a neuromuscular disease that is caused by mutations in the gene coding for polyA-binding protein nuclear 1 (PABPN1), which can lead to formation of intranuclear inclusion bodies causing muscle cell pathology. Patients with OPMD may have swallowing difficulties with potentially life-threating consequences, including malnutrition and aspiration pneumonia. OPMD is estimated to affect at least 15,000 patients in North America and Europe, and there are no products approved for treatment of the disease. AXO-AAV-OPMD is an adeno-associated viral (AAV) vector gene therapy delivered via a one-time intramuscular administration, which both silences the mutant PABPN1 gene and replaces it with a functional copy. The U.S. Food & Drug Administration and European Commission have granted Orphan Drug Designation to AXO-AAV-OPMD for the treatment of OPMD.
Under the terms of the agreement, Axovant will pay Benitec an upfront payment of $10 million for rights to the AXO-AAV-OPMD program and five additional investigational gene therapy products, as well as payments tied to development, regulatory and commercial sales milestones. In addition, Benitec will receive 30% of the net profits on worldwide sales of AXO-AAV-OPMD and tiered royalties on the other gene therapy products that result from this collaboration. The first additional investigational gene therapy product will target the C9orf72 gene, which is associated with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
“This expansion of Axovant’s pipeline further demonstrates our commitment to advancing innovative gene therapies for serious neurological diseases,” said Pavan Cheruvu, MD, chief executive officer of Axovant. “OPMD is a debilitating, potentially fatal disease that affects adults in the prime of their careers, and no approved treatment options are currently available. Quality-of-life in patients with OPMD can be impaired due to proximal muscle weakness, swallowing difficulties, aspiration pneumonia and malnutrition, and no approved treatment options are currently available. AXO-AAV-OPMD directly targets the underlying genetic defect that causes this disease using Silence-and-Replace technology, and I am excited about the potential of our gene therapy program for patients suffering from OPMD.”
“The Silence-and-Replace technology is a unique approach in gene therapy, using a single vector to suppress mutant protein production while also restoring expression of the functional protein, and could be an elegant solution to tackling autosomal dominant genetic disorders,” stated Fraser Wright, PhD, chief technology officer of Axovant. “I look forward to collaborating with the research and manufacturing teams at Benitec to advance the progress of the platform and bring additional therapies into the clinic.”
Commenting on the agreement, Jerel Banks, MD PhD, executive chairman of Benitec Biopharma said, “This agreement with Axovant further demonstrates the importance of Benitec’s core technology and our strategic focus on rapidly progressing these programs into the clinic. We believe Axovant is the ideal partner to take these programs forward, and look forward to working closely with them to develop AXO-AAV-OPMD and other neurological gene therapies.”
The AXO-AAV-OPMD Program is an investigational gene therapy being developed as a one-time treatment for oculopharyngeal muscular dystrophy (OPMD). The Program utilizes an AAV vector to silence the mutant poly-A binding protein N1 (PABPN1) gene that causes OPMD and replace with a functional copy of the PABPN1 gene. The Silence-and-Replace approach aims to knock down the expression of the mutant PABPN1 gene through ddRNAi, while at the same time express a re-engineered copy of the PABPN1 gene coding for the functional PABPN1 protein. The gene therapy will be delivered in a single administration directly into target muscle tissue to provide long-term correction of muscle pathology and restoration of function.
Data from mouse models of OPMD showed gene therapy from the AXO-AAV-OPMD Program provided up to 86% inhibition of PABPN1 expression, while restoring functional PABPN1 up to 63% of normal levels. The A17 mouse model is a well validated in vivo model that is designed to exhibit many of the key pathological features of OPMD patients. The levels of gene silencing and expression achieved in this model coincided with decreased muscle pathology and a restoration of muscle force and muscle weight to near wild-type levels.
Axovant expects to initiate a placebo-controlled clinical study for the investigational AXO-AAV-OPMD Program in 2019. The US Food & Drug Administration and European Commission have granted Orphan Drug Designation to the AXO-AAV-OPMD Program for the treatment of OPMD.
Oculopharyngeal muscular dystrophy (OPMD) is a muscular disease that is inherited through a primarily autosomal dominant pattern. OPMD is estimated to affect approximately 15,000 people in North America and Europe. The disease generally presents between the ages of 40-70 years old and is characterized primarily by progressive swallowing difficulty (dysphagia), eyelid drooping (ptosis), and weakness of the proximal extremities. Swallowing difficulties can have life-threating consequences, including malnutrition and aspiration pneumonia. As the disease progresses, the dysphagia becomes more severe and other muscles may become involved. There are no products approved for the treatment of OPMD and therefore, treatment options available to patients are limited.
OPMD is caused by mutations in the gene coding for polyA-binding protein nuclear 1 (PABPN1), a ubiquitously expressed protein that regulates the processing of messenger RNAs. The normal PABPN1 protein contains ten copies of the amino acid alanine, which forms a polyalanine tract. In OPMD, the mutated PABPN1 gene has an expansion of alanine-encoding trinucleotide repeats, resulting in an abnormally long polyalanine tract. The protein that forms from the mutated gene is prone to aggregating into insoluble nuclear inclusion bodies which leads to muscle cell pathology and disease progression.
In order to defeat the menace of counterfeit medicines and develop greater standards of healthcare and food packaging, Svam Packaging Industries Pvt Ltd is partnering with Japanese packaging giant- Toyo Aluminium K.K., the world’s finest technology driven aluminium company established since 1931. This is amongst the biggest FDI in Indian healthcare packaging industry. This partnership is in alignment with government of India’s ‘Make in India’ initiative and would help create employment in the country.
As per the agreement, Toyo Aluminium K.K. will acquire 33.4% equity in Svam Packaging Industries Pvt Ltd and bring in its advanced Japanese technology in the packaging sector and also focus on anti-counterfeiting solutions to control the problem of spurious drugs and medicines.
In the healthcare industry, cold form (Alu Alu) is the most effective form of packaging for heat sensitive drugs requiring moisture and gas barrier properties. It is widely used and fastest growing product in the packaging segment globally in the pharmaceutical sector. Svam currently has about 1/3rd market share of Indian cold form industry and caters to nearly all the leading pharmaceutical companies of India. Apart from India, the company exports to countries such as Europe, South America, Middle East, South Asian countries and many more. The company has state of art facility with to manufacture over 10,000 tons of cold form foils annually to meet the growing needs of pharmaceutical industry.
Toyo Aluminium, established since 1931, is the biggest aluminium packaging company headquartered in Japan with a turnover of nearly a billion dollars, has a strong presence across several countries such as Europe, USA, China and many more. The company is now investing in India in partnership with Svam to expand its wings now in the packaging sector. The company has several patents and specializes in research of aluminium products.
Through this partnership, the two companies will leverage each other’s strength in R&D and efficient human resource. Svam Toyal will manufacture innovative aluminium foil packaging solutions such as laminates for pharmaceutical and food packaging industry. The company would set up manufacturing units both within and outside India and offer innovative products in healthcare, food and beverage packaging segment. The main manufacturing unit and central hub would be within India itself. The company intends to increase production on a massive scale of the cold form (Alu Alu) foil as well as other products such as special aluminium lidding foils, child resistant laminates, Toyal Lotus, specialised printing applications and other cost effective solutions for the packaging industry.
Satish Ailawadi, managing director at Svam Packaging Industries Pvt Ltd said, “We are privileged to partner with Toyo Aluminium K.K. who are world leaders in creative and innovative packaging products. This partnership will help us serve our customers better and provide safer packaging products in healthcare as well as food and beverage industry.”
“Our team continuously monitors new trends in the packaging industry to develop special features that can add value to the products, brands and anti-counterfeiting solutions and also cultivates new cost effective laminates to ensure finest packaging for the pharmaceutical industry,” added Satish Ailawadi.
Speaking on the occasion, Hiroshi Yamamoto, president, Toyo Aluminium K.K. said, “India is a growing market with requirement of innovative products. We want to invest in India’s remarkable growth story and ensure product safety through offering our innovative technology in the packaging domain.”
“We are proud of the fact that we are able to offer the high-level technological solutions that we have developed over many years to produce pioneering products which are unique in the industry. We will continue our advanced research and development to unravel the infinite possibilities of aluminium so that we can make products safer for the end consumer,” Hiroshi Yamamoto said.
Lancet Gastroenterology & Hepatology publishes results from two Goofice phase 3 studies in chronic constipationRead Now
Eisai Co., Ltd., its subsidiary for gastrointestinal diseases EA Pharma Co., Ltd. and Mochida Pharmaceutical Co., Ltd. announced that results from two phase 3 clinical trials (a 2-week double-blind placebo-controlled phase 3 trial and an open-label single-arm 52-week long-term phase 3 trial) for the bile acid transporter inhibitor “Goofice 5mg tablet” (nonproprietary name: elobixibat hydrate; development code: AJG533, hereinafter 'elobixibat') have been published in The Lancet Gastroenterology & Hepatology), a journal of The Lancet which is one of the world’s most influential medical journals.
The 2-week double-blind clinical trial was a placebo-controlled, randomized, double-blind trial with 133 Japanese patients with chronic constipation. Patients were orally administered 10 mg of elobixibat or placebo once daily for 2 weeks. The elobixibat group demonstrated statistically significant improvements in the primary endpoint of change in spontaneous bowel movement frequency, as well as in secondary endpoints including change in complete spontaneous bowel movement) frequency (the secondary endpoint), length of time between dosing and the first spontaneous bowel movement, compared to the placebo group. The major side effects were abdominal pain, diarrhea and other gastrointestinal symptoms. There were no serious side effects.
The 52-week open-label phase 3 clinical trial was a single arm trial to evaluate the efficacy and safety of long-term administration of elobixibat in 341 Japanese patients with chronic constipation. The initial dose was 10 mg once daily orally for 7 days. The dose was increased or decreased in the range of 5, 10 and 15 mg per day appropriately depending on the symptoms and given for 52 weeks. As a result, constipation-related improvements including spontaneous bowel movement frequency, complete spontaneous bowel movement frequency and stool consistency were observed as early as 1 week of administration, and the effects were maintained favorably through 52 weeks. The longer the dosing period, the higher patients’ satisfaction relating to defecation tended to be. In addition, in all the JPAC-QOL scores, there were statistically significant declines compared to baseline. The major side effects were abdominal pain, diarrhea, lower abdominal pain and other gastrointestinal symptoms. A serious side effect (inguinal hernia) was observed in 1 patient.
The above clinical trial results were presented at Digestive Disease Week (DDW) 2018, June 2-5 in Washington D.C., USA.
Elobixibat was jointly developed by EA Pharma and Mochida. EA Pharma and Mochida distribute elobixibat under the same brand name “Goofice 5mg tablet”, respectively, in Japan. EA Pharma and Eisai jointly provide proper use information of “Goofice 5mg Tablet” under a co-promotion agreement.
EA Pharma, Eisai and Mochida strive to make a further contribution to improve QOL for patients with chronic constipation through maximization of the product value of “Goofice 5mg tablet”.
SPDS organises 'Disso India--Hyderabad 2018' to introduce new technology and innovation related to dissolutionRead Now
Society for Pharmaceutical Dissolution Science (SPDS) conducted its 6th Annual International Convention 'Disso India--Hyderabad 2018' on June 28 and 29, 2018 at Hotel Avasa, Hyderabad. The event promoted introduction of new technology, innovation and discussed various issues related to dissolution. The two-day event witnessed presence of several eminent professionals from the pharmaceutical industry.
Disso India is the flagship International Conference of SPDS conducted every year and is attended by professionals from Indian pharma, R&D, QA and QC as well as the academia. Disso India-Hyderabad 2018 had 18 lectures from eminent professionals from pharmaceutical industry from around the world as well as the academia. Around 250 delegates from industry and academia participated in this conference.
The conference was organised under the chairmanship of Udaya Bhaskar, Director General, Pharmexcil, Hyderabad and the organizing secretary, Dr. B.M. Rao, Vice President and Head of corporate QC, Dr. Reddy’s Labs, Hyderabad.
The scientific sessions were programmed and executed under the chairmanship of Prof. Padma V. Devarajan, Professor in Pharmacy, Department of Pharmaceutical Sciences and Technology, Institute of Chemical Technology.
The event comprised of plenary lecture, panel discussion, exhibits and poster session.
Prof. Raghuram Rao Akkinepally, director – NIPER SAS Nagar who was the chief guest of the event inaugurated the event with the lighting of the event. The scientific abstract book was released at the hands of Dr. Raghuram Rao during the event.
Disso India - Hyderabad 2018 had exceptional global speakers who presented on varied topics.
While Dr. Anant Ketkar, Sun Pharma Advanced Research Company, India gave his presentation on PBPK Modelling and Simulation: An In silico - In Vivo Bridge for Efficient Formulation Development; Dr. Chuei Wuei Leong, Founder and Principal consultant, CEXA Consultancy Sdn Bhd, Malaysia delivered his speech on Pro-drugs Challenges In Dissolution. Dr. Namita Tipnis, University of Connecticut, USA gave a presentation on Application of USP 4 in dissolution testing of complex parenterals and IVIVC.
Other speakers who delivered speech in the event included Dange Veerpaneni, President/CEO, Sparsha Pharma, Hyderabad, India on challenges and development of trans dermal patches in perspective of dissolution studies; Grove Geoffrey, Product Manager and Application Scientist, SOTAX Corporation, USA on new developments in fully automated dissolution technology; Prof. Imre Klebovich, Professor of Pharmaceutics, Semmelweis University Department of Pharmaceutics, Budapest, Hungary on tracking the effect of drug interactions with in vitro dissolution studies; Dr. Jennifer Dressman, Prof. of Pharmaceutical Technology, Institute of Pharmaceutical Technology Biocenter, Johann Wolfgang Goethe University, Germany On the future of bioequivalence: changing paradigms at the FDA; Dr. Kailas Thakker, COO & Co-founder, Tergus Pharma, USA on IVRT for topical dosage forms as BA/BE waiver; Dr. Kalyanaraman; Senior Director – AR&D, Dr. Reddy’s Laboratories Ltd, India on opportunities & practical challenges in dissolution testing of generic drugs; Rashi Kochhar, Shimadzu India on chromatography automations in the QC lab; Dr. Roger Willams, Partner, NDA Partners LLC, USA on recent drug regulation and the pharmacopoeias; Saji Thomas, Director, Par Pharmaceutical, USA on life cycle management of analytical methods; Samir Haddouchi, Managing Director, SPS Pharma Services, Orleans, France on In vitro testing for non-conventional dosage forms; Dr. Sandip Tiwari, Fellow, Manufacturing Science and Technology, Actavis Laboratories FL, Inc., Florida, USA On dissolution failures: interesting scenarios; Seema Trivedi,GM Technical; Anshul Life Sciences, India on excipients quality & trouble shooting; Prof. Umesh Banakar, Professor and President, Banakar Consulting Services, USA On predicting bioavailability from dissolution: unlocking the mystery(ies); Vijay Kshirsagar, Director and CEO, TRAC Pharma Consulting, Mumbai, India On current regulatory observations & trends related to dissolution studies; and Dr. Vinod Shah, Ex. US FDA, Pharmaceutical Consultant, USA on a science based approach to simplify regulatory pathway for a complex generics using IVRT.
SOTAX India was the platinum partner of this event. Other partners were Novartis, Electrolab, Mylan, ACG, Inveniolife, Labindia, Shimadzu, Anshul Life Sciences, Pharmexcil and USP. There were 14 selected posters which were presented during this event out of which the best 3 were awarded by Sotax India.
Dr. Reddy's Laboratories Limited and UCB, a biopharmaceutical company, announced that they have entered into a distribution and co-promotion agreement for Briviact, a brand of brivaracetam. The agreement grants Dr. Reddy’s the exclusive right to distribute Briviact in India.
Briviact (brivaracetam) is approved as an adjunctive therapy for the treatment of partial-onset seizures in epilepsy patients who are 16 years of age and older.
MV Ramana, CEO - Branded Markets (India and Emerging Markets), Dr. Reddy’s said, “In our endeavour to make innovative medicines accessible to patients in India, we are excited to partner with UCB India for Briviact, a novel treatment for epilepsy that will make a difference to the lives of patients living with epilepsy.”
“We know that as many as one third of people with epilepsy are currently uncontrolled on their existing medicines,” explained Max Bricchi, head of International Markets, UCB Neurology Patient Value Unit. “This partnership is another important step towards us providing value together to patients by making Briviact available as an additional treatment choice for epilepsy.”
Epilepsy is the second most common neurological condition and as per a recent study, an estimate of 70 million people suffer from it worldwide. There are over 12 million people suffering from epilepsy in India, which contributes to nearly one-sixth of the global burden.
Hansa Medical, a biopharmaceutical company developing novel immunomodulatory enzymes for transplantation and acute autoimmune disease, announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to imlifidase (IdeS) for the treatment of anti-GBM antibody disease (anti-GBM), also known as Goodpasture's disease.
Anti-GBM is a severe kidney disease where the immune system mistakenly develops IgG-antibodies, resulting in an acute immune attack on the kidneys and in some patients also on the lungs. In severe anti-GBM, the disease may progress to renal failure or death. There is no approved treatment of anti-GBM and less than one third of the patients survive with a preserved kidney function after six months follow-up.
“We believe that imlifidase’s fast, effective and well tolerated IgG eliminating property has the potential to make a significant difference for patients with anti-GBM”, said Søren Tulstrup, CEO at Hansa Medical AB. “We are very pleased to receive orphan designation for imlifidase in the US for the treatment of anti-GBM. This both confirms the high unmet medical need and further encourages us to continue our clinical investigations with imlifidase in this devastating disease.”
The FDA Orphan Drug Act (ODA) provides for granting special status to a drug or biological product to treat a rare disease that affect fewer than 200,000 people in the US. Orphan drug designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits, protocol assistance and up to seven years of US marketing exclusivity from time of approval of BLA.
In June 2017, an open label investigator-initiated phase II study in severe anti-GBM antibody disease (ClinicalTrials.gov identifier NCT03157037) was initiated with Hansa Medical lead candidate imlifidase with Professor Ma°rten Segelmark at Linko¨ping University Hospital, Sweden, as coordinating principal investigator and sponsor. The aim is to enrol approximately 15 patients in Sweden, Denmark, Austria, Czech Republic, France and the UK by the end of 2018. The primary objective of the study is to evaluate the safety and tolerability of imlifidase in patients with severe anti-GBM antibody disease in addition to standard-of-care. Imlifidase efficacy will be assessed by evaluating renal function at six months after imlifidase treatment.
Creo Medical Group plc, a medical device company focused on the emerging field of surgical endoscopy, announced that it has entered into a framework distribution agreement with First Medical Company (First Medical).
Under the terms of the agreement, First Medical in conjunction with Creo will seed the South Africa, Botswana, Namibia and Mozambique markets with Creo’s CROMA advance energy platform and Speedboat products over an 18 month period. First Medical will purchase a minimum quantity of CROMA advance energy platforms and Speedboat instruments during the first 18 months. In line with Creo’s education led commercial strategy, First Medical will also advance the roll out of Creo’s Clinical Education Program in its jurisdictions, to ensure quality control and best patient outcomes through the education of key clinicians in the use of Speedboat and the CROMA platform. On completion of the seeding phase, it is anticipated that a further agreement will place First Medical as Creo’s exclusive distributor for Creo’s CROMA advanced energy electrosurgery platform and Creo’s suite of GI devices in South Africa, Botswana, Namibia and Mozambique.
The first CROMA advance energy platforms and Speedboat products for clinical use in South Africa, Creo’s first export market in the EMEA region, have been shipped to First Medical, and market seeding is expected imminently.
Founded 20 years ago, First Medical imports and distributes medical products and devices into Southern Africa. It has developed strong and long-term relationships with key clinicians in the region, which will assist in establishing Creo’s Clinical Education Program in Southern Africa. First Medical has more than 300 clients ranging from medical professionals to both private and government hospitals. They are a leading company in the field of endoscopy.
Creo’s CROMA’s electrosurgical platform combines bipolar radiofrequency for precise localised cutting and microwave energy for controlled coagulation. Speedboat RS2 is the first product approved in a suite of tools under development to aid the endoscopist in minimally invasive surgery. The Speedboat device helps reduce the risks associated with alternative open and laparoscopic procedures, reducing the length of hospital stays, cost of treatment and transferring therapy from the operating theatre to the endoscopy room.
Craig Gulliford, Creo’s chief executive officer, commented,“This third agreement, alongside Hoya Group, Pentax Medical and Diagmed Healthcare, demonstrates the growing momentum in the commercialisation of our lead product, Speedboat RS2. With a rapidly growing distribution network, Creo is well placed to advance the emerging field of surgical endoscopy through our advanced energy platform, paving the way for our suite of minimally invasive GI devices under development. First Medical’s expertise and knowledge of the Southern African markets will prove invaluable to Creo as we continue to build our reputation worldwide.”
Axonics Modulation Technologies, Inc., developer of the first rechargeable Sacral Neuromodulation (r-SNM) system for the treatment of urinary and bowel dysfunction, announced that it has received the CE mark for its Sacral Neuromodulation External Trial System.
Axonics received CE mark in June 2016 for its miniaturized implantable neurostimulator, tined lead, programmers, charger and related accessories. The External Trial System is an additional element of the Axonics r-SNM System used to help identify responders to Sacral Neuromodulation therapy prior to a permanent implant. It is composed of a temporary, single-use disposable external stimulator that is connected to either a Tined Lead or a temporary Peripheral Nerve Evaluation (PNE) Lead, depending on the preferred trial method.
This EU regulatory approval coincides with the commercial launch of the Axonics r-SNM System in the United Kingdom. Additional regulatory approvals in Europe are expected in the near term, including full-body MRI conditional labeling.
Axonics is currently conducting a 120-patient pivotal clinical study under a US Food & Drug Administration (FDA) Investigational Device Exemption for urinary dysfunction and on June 27, 2018, announced completion of the enrollment and implant phase. The company anticipates FDA approval in the United States after the 6-month post-implant endpoint has been reached for all patients and the FDA has completed its review of the pre-market approval (PMA) application for the Axonics r-SNM System.
“To date, over 225 patients have been implanted with an Axonics r-SNM System in Europe and North America, the vast majority of which without undergoing an external trial period before permanent implant. We demonstrated that with quality lead placement and confirmation of intraoperative responses, outstanding clinical results can be achieved without an external trial,” said Raymond W. Cohen, chief executive officer of Axonics. “However, from a commercial standpoint, having an External Trial System is necessary given that payors prefer to reimburse permanent implants after confirming that the patient is a trial responder. We are confident that Axonics is now well positioned to capture significant worldwide share and, due to the benefits of our product offering, accelerate growth in the large $700 million Sacral Neuromodulation market.”
In April 2018, Axonics raised its latest round of capital to support commercialization activities in Europe and in the US. Since March 2014, with the support of a number of the world’s highest profile venture firms located in the US, France, the United Kingdom, the Netherlands, Switzerland and China, Axonics has raised over $114 million in equity capital in addition to a $20 million venture loan from Silicon Valley Bank.
Overactive bladder affects an estimated 85 million adults in the US and Europe. Another 40 million are reported to suffer from fecal incontinence. SNM therapy is an effective and durable treatment that has been widely used and reimbursed in Europe and the US for the past two decades. It is estimated that well over 300,000 patients have benefited from the therapy to date. SNM is the only OAB treatment with proven clinical superiority to standard medical therapy and OAB patients who receive SNM report significantly higher quality of life than patients undergoing drug treatment.
Since, family planning has been enunciated as a human right in the UN Charter, reiterated in all international and regional human rights agreements, and now brought to focus by the theme for World Population Day 2018, ‘Family Planning is a Human Right’, according to ARC coalition statement to mark the celebration of World Population Day 2018 on 11 July.
Access to contraceptive information is fundamental to achieving gender equality and we need to stress the importance of male engagement for a successful family planning programme, according to ARC coalition statement.
Advocating Reproductive Choices (ARC) is a coalition of more than 170 civil society organisations and individuals that are committed to advocating for greater attention and focus on sexual and reproductive health issues and family planning services in India. Established in 2005, the coalition aims to expand contraceptive choices and call for greater attention to the quality of care of family planning services for the Indian population. ARC is comprised of organisations that have technical ability and implementation expertise in reproductive health and family planning.
ARC makes concerted and sustained advocacy efforts to enhance accessibility and expand contraceptive choices available to all women in India. The ARC Core Committee oversees strategic decisions and plans made by the coalition. There are five state chapters of ARC in Rajasthan, Uttar Pradesh, Madhya Pradesh, Bihar and Jharkhand. In 2005 its first Secretariat was with Parivar Seva Sansthan (PSS). In 2007 Family Planning Association of India became the next Secretariat. Since, 2015 Population Foundation of India is holding the ARC Secretariat.
India’s commitment at the FP2020 platform to expand access to Family Planning services has also provided an impetus to FP, resulting in the introduction of new contraceptives and progrmmes such as the Mission Parivar Vikaas to increase the availability and accessibility of commodities and services. However, with approximately 13 per cent of currently married women between the ages of 15 and 49 who have an unmet need for spacing or limiting births, India has the largest number of women with unmet need for contraception in the world.
The International Conference on Human Rights held in 1968, was the first to embed family planning in legislative language when they stated, “Parents have a basic human right to determine freely and responsibly the number and spacing of their children.”
India holds the legacy of introducing the first ever family planning programme in 1952. However, it was only in 1994, when India became a signatory to the International Conference on Population and Development held in Cairo, that we shifted focus to a rights-based approach that valued people and their welfare more than meeting a population target. Since then there have been several changes in the policy and programmes for Family Planning in India.
Access to family planning has been associated with women’s empowerment and advancing women’s status in society; women’s literacy, age of marriage, and participation in the workforce which contribute to their decision making powers. However, in India, the emphasis of family planning has been skewed against women with the focus being on female sterilisation. India’s reliance on modern contraceptives for spacing births is also low, with a 1.5 per cent uptake of IUDs, 4.1 per cent women reliant on the pill, and 5.6 per cent males who use condoms.
Furthermore, India's Family Planning program, based on demographic principles, focuses on married couples, and specifically on married women. There is a need for an enabling environment where every individual in the reproductive age group, irrespective of age or marital status, can openly seek and access quality family planning services. Availability, accessibility and quality of services need to be universally ensured to achieve the desired result. We must reinforce our existing strategies and foster innovations in the arena of family planning, while keeping in mind an individual’s right to choose whether, when and how many children to have.
More importantly, there is also a need to expand the definition of Family Planning is broader beyond only contraception. The right to choices and treatment options that help an infertile couple to have a baby are yet not a part of primary health care.
Therefore, the Family Planning programme needs to be broad based to fulfill these rights. This implies a great deal of responsibility on the Ministry of Health and Family Welfare, and equally on civil societies and private sector to play a catalytic role in ensuring provision of quality FP services and empowering the present and future generation in their decision making process.
Hikma Pharmaceuticals PLC announced that it has signed a licensing and distribution agreement with Omega Pharma Trading NV, an affiliate of Perrigo Company plc (Perrigo), one of the largest providers of over-the-counter healthcare solutions in Europe.
Under the terms of the agreement, Hikma has the exclusive right to license and distribute more than 30 consumer healthcare products, including Davitamon, Prevalin, XLS Medical, Dermalex and Paranix, in all its MENA markets, with the exception of current agreements in place. Hikma aims to work with Perrigo’s existing partners in the MENA region to broaden sales and marketing coverage.
In addition, Hikma has the right of first refusal to the full range of Perrigo’s OTC medicines in the region.
The partnership provides a significant opportunity for Hikma to collaborate with Perrigo in MENA, building on the strengths of each company. The agreement will leverage Hikma’s substantial local presence with operations in 17 markets and an established sales and marketing team, experienced in building brands, with particular expertise in the consumer health business.
Mazen Darwazah, executive vice chairman, chief executive of MENA and Emerging Markets said, “We are very pleased to be entering a partnership with Perrigo. This agreement will expand our consumer healthcare portfolio, enabling us to meet growing demand across the region.”
Svend Andersen, Perrigo executive vice president and president, Consumer Healthcare International, stated, “These OTC medicines are important in meeting the healthcare needs of patients and consumers. We are pleased to partner with Hikma to leverage their strong presence in MENA markets, further increasing access to these valuable consumer products.”
NBHA joins hands with 70 organizations globally to support fragility fracture network's call to actionRead Now
The statistics on osteoporosis-related bone breaks are alarming and will only get worse as the Baby Boomer population continues aging. Currently someone in the US breaks their hip every two minutes. It costs $19 billion annually to treat fractures among seniors (a figure projected to increase to over $25 billion by the year 2025), yet the majority of patients are released without being evaluated for osteoporosis — the underlying disease which may have led to the fracture.
In effort to reverse the current trend, the National Bone Health Alliance(NBHA) is joining more than 70 leading organizations from around the world in supporting a Global Call to Action to improve care for people who suffer fragility fractures. Published online by Science Direct, the Call to Action outlines transforming the surgical and medical care provided to those hospitalized with a hip fracture, spinal fracture or other major fragility fracture; efforts to prevent second and subsequent fractures following a person's first fragility fracture; and rehabilitation to restore mobility and independence for people whose ability to function is impaired after a hip or other major fragility fracture.
"It is no longer acceptable for patients to be treated for fractures and released from medical care without assessment and interventions to reduce their risk of a repeat fracture," said Susan Greenspan, MD, Professor of Medicine and Director, Osteoporosis Prevention and Treatment Center, University of Pittsburgh, NOF President and NBHA Co-Chair. "The Fragility Fracture Network's Call to Action supports NBHA's ongoing commitment to secondary fracture prevention and we're thrilled to see the international community recognizing the need to transform the treatment and rehabilitation people receive after suffering a fragility fracture."
"After suffering an osteoporotic fracture, a patient's likelihood of suffering another fracture increases three to five times, yet currently only about a quarter of patients are evaluated and treated for osteoporosis after their first fracture," said Dr. Robert Adler, Professor of Internal Medicine, Epidemiology and Community Health, Virginia Commonwealth University School of Medicine, Chief, Endocrinology and Metabolism, McGuire Veterans Affairs Medical Center, and NBHA Co-Chair. "With our commitment to secondary fracture prevention and the support of this Global Call to Action, we can close the current 70 to 80 percent care gap for testing and treating patients over age 50 who experience a fracture."
To tackle the current osteoporosis crisis, six leading organizations recently came together at an annual congress of the fragility fracture network to develop the global call to action on fragility fractures.
"Fragility fractures can devastate the quality of life of people who suffer them and are pushing our already overstretched health systems to a breaking point," said Professor Karsten E. Dreinhöfer, lead author of the publication. "As the first of the baby boomers are now into their seventies, we must take control of this problem immediately before it is too late."
The Global Call to Action illustrates that for the first time, all the leading organizations in the world have recognized the need for collaboration on an entirely new scale. The Global Call to Action proposes specific priorities for people with fragility fractures and their advocacy organizations, individual health workers, healthcare professional organizations, governmental organizations, insurers, health systems and healthcare practices, and the life sciences industry.
AbbVie and Celgene are pharma's two biggest losers in terms of clinical development this year. But that doesn't mean investors should shy away from these two names.
Developing new drugs is wildly expensive. Since 2007, for instance, the average cost of bringing a novel drug to market (a new molecular entity) has hovered around a jaw-dropping $3.9 billion, according to EvaluateGroup. When all drugs are thrown into the mix, however, this figure balloons to an unreal $5.5 billion.
The point here is that it's extremely painful for big pharmas and blue-chip biotechs when their star clinical candidates flame out in late-stage testing, or suffer a serious regulatory setback. Even so, clinical and regulatory setbacks happen all the time in the pharma industry. Investors therefore need to have a solid understanding of how these setbacks impact valuations in the sector from both a short- and long-term perspective.
With this theme in mind, here is a look at how AbbVie (NYSE:ABBV) and Celgene Corporation (NASDAQ:CELG) made the two most costly mistakes halfway through 2018 and why investors in these two elite biopharmas shouldn't necessarily panic over these blunders.
1. AbbVie's Rova-T
In June 2016, AbbVie doled out $5.8 billion to acquire Stemcentrx for its cancer stem-cell therapy called Rova-T (rovalpituzumab tesirine) as a possible game-changing treatment for small-cell lung cancer. Immediately thereafter, analysts chimed in to name Rova-T as one of the most valuable experimental therapies in the clinic at the time. EvaluateGroup, for example, had the therapy's peak sales coming in at a staggering $8.3 billion by 2023, which would have propelled it into the top five best-selling pharma products in the world.
What a difference two years make. After posting dismal midstage trial results for third-line small-cell lung cancer last March, AbbVie revealed at the recent American Society for Clinical Oncology meeting that Rova-T's ongoing trials are showing little sign of efficacy, and poor side-effect profiles to boot. The point being that Rova-T went from a shining star to a potential complete dud in a year's time. As a result, EvaluateGroup downgraded its outlook for the drug's peak sales to a mere $193 million by 2024. That's a stunning 98% drop from where the drug stood in terms of market value back in 2017.
How did this setback impact AbbVie's valuation? Over the course of Jan. 1, 2017, to the day before Rova-T's midstage results were released in March 2018, AbbVie's shares were up by 80%. That kind of high-flying action is certainly unusual for a large-cap biotech stock, perhaps reflecting the market's enthusiasm for this key pipeline asset. In the intervening three-plus months since Rova-T's initial data release, however, AbbVie's shares have been extremely volatile, shedding nearly 7% of their value overall. That's not the end of the world, but AbbVie's upward momentum has certainly tapered off noticeably.
2. Celgene's ozanimod
Approximately three years ago, Celgene purchased Receptos for a noteworthy $7.2 billion for its experimental immunomodulatory drug ozanimod. At the time, the drug was already in human trials for both multiple sclerosis and ulcerative colitis; it had shown promise as a truly game-changing therapeutic option for these hard-to-treat conditions. Celgene thus rolled out a peak sales estimate for ozanimod of between $4 billion to $6 billion during its initial press release announcing the acquisition.
The take-home point here is that with this pricey acquisition, the blue-chip biotech appeared to have finally found its heir apparent to the aging multiple-myeloma drug Revlimid as its next flagship product.
Then the wheels fell off. Despite strong late-stage trial data showing that ozanimod was indeed a potent new therapy for relapsing multiple sclerosis, Celgene reportedly allowed the skeleton crew remaining at the Receptos facility in California to handle the drug's regulatory application with the Food and Drug Administration (FDA). At least that's what Celgene said after the drug got nailed with a refusal-to-file letter from the FDA for missing preclinical and clinical pharmacology data last February. Now the drug isn't scheduled to reach the market until perhaps late 2019.
Why is this regulatory delay such a big deal? Analysts believe that this lengthy delay could allow the drug's competitors across its various indications to catch up from a development standpoint. If so, these same analysts think that ozanimod's commercial opportunity could be slashed by a stunning 56% from peak. That's seriously bad news, especially as the company searches for a way to keep growing beyond Revlimid's eventually date with the patent cliff.
So it's not surprising that Celgene's shares dropped by as much as 25% from their 52-week highs in the months following this self-inflicted wound.
Can these biopharmas adapt and overcome?
Although investors were none too pleased with these setbacks, the good news is that both AbbVie and Celgene have vibrant pipelines capable of making up for lost ground. As proof, AbbVie and Celgene have the second and third most valuable clinical pipelines, respectively, across all of biopharma, according to EvaluateGroup.
That's not surprising given that both companies have taken an exceptionally aggressive approach to pipeline development through external licensing deals, as well as mergers and acquisitions over the past few years.
The net result is that the shares of both AbbVie and Celgene are arguably a great bargain here. AbbVie's top line is still forecast to rise at a healthy 4% compound annual growth rate (CAGR) over the next five years, despite losing exclusivity for its top-selling anti-inflammatory drug Humira and Rova-T's implosion. And Celgene is expected to perform even better over this same period with a stately 9% CAGR. Of course, these growth forecasts would have been richer if Rova-T and ozanimod didn't get derailed, but that doesn't mean the sky is falling, either.
The bottom line is that AbbVie and Celgene prepared for the possibility of a serious setback by building out extremely robust pipelines capable of smoothing out the rough spots over the long term.
Interesting enough, though, several other large biopharmas such as GlaxoSmithKline and Sanofi have failed to follow this same prudent course of action during the height of the patent cliff by relying way too heavily on a small set of experimental product candidates to deliver long-term growth. As such, their shares have drastically underperformed the broad biopharma space over the last decade.
AbbVie and Celgene, on the other hand, apparently learned from the follies of their peers by building pipelines with multiple shots on goal, so to speak. And that's the kind of deep value that savvy investors should appreciate.
AbbVie is not on our top "Buy" list, but these 10 stocks are
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Broad Institute of MIT, Bayer partner to usher in innovative technologies to address heart failure and pursue new drug targetsRead Now
The Broad Institute of MIT and Harvard and Bayer are launching the Precision Cardiology Laboratory (PCL), a new endeavor that will pursue scientific insights aimed at developing new therapies for heart failure.
According to the American Heart Association, more than 900,000 people are diagnosed with heart failure every year in the United States. The condition, which results from the failure of the heart to pump enough blood, is one of the most common reasons for hospitalization among adults. There are many causes of heart failure, and the PCL will use new tools and methods to more fully understand and treat them.
Research in the PCL will bring scientists from both organizations into an integrated work space at the Broad Institute, effectively combining Broad's innovative methods for basic scientific discovery and the clinical expertise of its practicing physician/researchers with Bayer's long history of drug development. The effort will be led by Broad Associate Member Patrick Ellinor, who directs the Cardiac Arrhythmia Service at Massachusetts General Hospital and is a professor of medicine at Harvard Medical School.
The PCL's initial goal is to develop high-resolution, single-cell maps of cardiovascular tissues in human and animal models. Using tissue samples donated by healthy individuals as well as people suffering from cardiovascular disease, researchers will build datasets to accelerate insights into heart failure.
"Such high-resolution maps of cells and tissues will be a profound asset for understanding heart failure and for developing new and better drugs," said Ellinor. "I am extremely excited by the potential of this expanded partnership to benefit patients."
The Broad-Bayer partnership began in 2013 with an oncology program. In 2015, the organizations launched a cardiovascular-specific collaboration aimed at using genomics to better understand cardiovascular disease. Now, the PCL will use non-genomic approaches to jumpstart the development of new therapeutics for heart failure.
Through this expanded partnership, Bayer is dedicating an additional $22 million to the collaboration over the next five years.
"The Broad Institute is an important and strategic partner for Bayer enabling us to deepen our understanding in the area of cardiovascular diseases and we are looking forward to extending our collaboration even further," said Dr. Joerg Moeller, member of the Executive Committee of Bayer AG's Pharmaceuticals Division and Head of Research and Development. "Joint laboratories are a novel partnering model for industry and academia and will bring Bayer and Broad Institute cardiovascular research to the next level."
The PCL will be guided by a joint steering committee. Ultimately, the lab will involve roughly 20 people with affiliations divided between the two organizations. The institutions will continue to openly share findings that arise from the collaboration through both publicly-available datasets and academic journals.
Broad Institute of MIT and Harvard was launched in 2004 to empower this generation of creative scientists to transform medicine. Bayer is working in a wide range of therapeutic areas on new treatment approaches for cardiovascular, lung and kidney diseases. The cardiology franchise at Bayer already includes a number of products and several other compounds in various stages of preclinical and clinical development.
Bayer is a global enterprise with core competencies in the Life Science fields of health care and agriculture. Its products and services are designed to benefit people and improve their quality of life.
LabCorp, a leading global life sciences company that is deeply integrated in guiding patient care, and Royal Philips, a global leader in health technology, announced a collaboration to further advance digital pathology. Through the implementation of the Philips IntelliSite Pathology Solution, LabCorp will incorporate digitized workflows within its anatomic pathology services, which are an integral component of its comprehensive clinical laboratory and end-to-end drug development services.
Philips IntelliSite Pathology Solution is the first, and currently the only, digital pathology solution marketed for primary diagnostic use in the US. It aids pathologists in the review and interpretation of digital images of surgical pathology slides that are prepared from formalin-fixed paraffin embedded (FFPE) tissue, with the goal of providing improved turnaround times and supporting the precise analysis of tissue samples. LabCorp will initially implement the system in four of its laboratories, and it will also work with its interested customers to evaluate their potential transition to digital pathology.
“Digital pathology opens new, innovative ways to help laboratories and hospital systems improve workflows and provide better patient care,” said Marlon Thompson, General Manager of Philips Digital Pathology Solutions. “Together with LabCorp, we can accelerate the digital transformation of pathology and intensify collaboration between laboratories and healthcare professionals.”
“Digital pathology offers the opportunity for improved efficiency and enhanced collaboration between our pathologists and our customers,” said Gary M. Huff, CEO of LabCorp Diagnostics. “We are pleased to be the first major commercial laboratory to implement the Philips IntelliSite Pathology Solution, and we look forward to working closely with Philips to identify the best way to integrate a digital pathology solution into our anatomic pathology workflow.”
Philips IntelliSite Pathology Solution is an automated digital pathology image creation, viewing and management system comprised of an ultra-fast scanner, an image management system and a display. This solution contains advanced software tools to manage the scanning, storage, presentation, reviewing, and sharing of images. By supporting the transition to digital workflows, Philips seeks to help pathology laboratories simplify access to histopathology information and implement more efficient workflows.
“Digital pathology is an emerging technology with the potential to improve the delivery of care for patients,” said Dorothy (Dot) Adcock, MD, chief medical officer for LabCorp Diagnostics“. The opportunity to more quickly access tissue images for analysis, to collaborate more readily with specialty pathologists around the globe, and to provide a faster diagnosis can improve patient care in often challenging areas of cancer diagnosis.”
Opioid addiction and abuse remains an immense public health crisis. We're committed to continuing to take new steps to address these protracted dangers from every possible angle.
A crisis of this magnitude requires broad, collaborative and creative approaches. We're especially aware that as we take more actions to curb the lawful prescribing of prescription opioids, our actions will inevitably push more and more people to obtaining opioids from illicit sources. And we know that digital drug dealers and other criminals are increasingly selling their illicit opioids through the internet, including social media and illegal online pharmacies. This activity is contributing to the public health emergency of opioid-related overdose deaths that have ripped apart families and communities. We aim to confront these new threats.
We know that the internet – both the surface and the dark web – aren't the only marketplaces for the illegal sale of prescription drugs. But they're perhaps the most far-reaching conduit for illicit drugs, and these new avenues present unique challenges for tech companies, law enforcement, as well as for the US FDA.
As we discussed yesterday, there's no gray area here: no controlled substances, including opioids, can be lawfully sold or even offered to be sold online. In the past, we've been one step behind the opioid epidemic. We cannot continue to be. We must stay one step ahead of this burgeoning crisis by frustrating and eventually stopping these digital drug dealers. We cannot ignore this space simply because other illicit sources of opioids, such as diversion, theft and smuggling, are still the more predominant routes by which people currently obtain illegal drugs. We're going to take new steps, and direct new investigative and criminal oversight resources, to stop the illegal sale of opioids online. But we cannot do it alone.
We appreciate the candid dialogue we had with summit participants, and their willingness to share information about the unique nature of their platforms, as well as existing surveillance and security protocols. While we agreed to safeguard some of the sensitive discussion points, especially to make sure that we don't tip off the bad actors who are closely watching our tactics and trying to stay ahead of our efforts, in the interest of transparency, I want to provide an overview of some of the main themes and takeaways from the presentations and roundtable discussion that took place throughout the day.
We heard from all the attendees about the important efforts they're taking to tackle this problem, and it's important that we acknowledge that positive steps are underway. We also discussed the various challenges that stakeholders viewed as potential barriers to take the next steps in some cases, from a lack of data that would help inform their approaches, to the potential policy and regulatory concerns.
Some of the approaches we heard during the summit were promoting legitimate or educational content in top search results or social media posts, and making it harder for individuals to find illegal sellers online. We also discussed ways to make use of big data and machine learning/artificial intelligence to identify illegal activity. And we heard about efforts to make public health resources more accessible – such as educational information about the risks of purchasing opioids online, and connecting individuals and family members to validated treatment programs to support those who are seeking a path toward recovery.
Noxxon updates on patient recruitment in ongoing NOX-A12 pancreatic and colorectal cancer clinical trialRead Now
Noxxon Pharma N.V., a biotechnology company focused on improving cancer treatments by targeting the tumor microenvironment (TME), announced that patient recruitment is almost complete in its ongoing clinical trial (NCT03168139) testing NOX-A12 alone (part 1) and subsequently in combination with Merck & Co./MSD’s Keytruda (part 2) in metastatic, microsatellite stable pancreatic and colorectal cancer patients.
Top-line data for part 1 of the ongoing clinical trial of examining the effects of NOX-A12 monotherapy on the tumor microenvironment is now expected to be available in the third quarter of 2018. Timing for top-line data from all patients in part 2 remains unchanged at Q4 2018.
Part 1 of the study compares tumor biopsies taken at baseline and after two weeks of NOX-A12 monotherapy in order to obtain data on the NOX-A12 mode of action in the tumor microenvironment. Interim data released in May 2018 showed markers consistent with Th1 type immune responses in a number of patients treated with NOX-A12 therapy alone. Additionally, changed levels of CXCL12 in tumors confirmed penetration of NOX-A12 into tumor tissue.
“The team at the National Center for Tumor Diseases in Heidelberg recently had several potential patients that were finally not eligible for our clinical trial,” said Aram Mangasarian, CEO of Noxxon. “As a result, we will need more time to complete recruitment. We will then analyze the data as per the trial design to extend the results already announced before the ASCO Annual Meeting in June 2018. We remain confident that we will be able to provide top-line data from part 2 of the trial on schedule in the fourth quarter of 2018.” Noxxon’s oncology-focused pipeline acts on the tumor microenvironment (TME) and the cancer immunity cycle by breaking the tumor protection barrier, blocking tumor repair and exposing hidden tumor cells.