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.