Orphan Drug Status
Orphan drug designation is granted to drugs intended for the treatment of life-threatening or chronically debilitating rare diseases where no therapeutic options are either authorized or where the drugs will be of significant benefit to those affected by the condition. Rare diseases are those defined as having a prevalence of no more than five in 10,000 persons in Europe or affecting less than 200,000 patients in the US.
The designation provides development and commercial incentives, including up to ten years of market exclusivity in the EU and up to seven years in the US, protocol assistance on the development of the drug, including clinical studies and certain exemptions from or reductions in regulatory fees. Hansa Medical has been granted the following orphan drug designations for imlifidase as of November 2018:
- Orphan drug designation by EMA of imlifidase for the prevention of graft rejection following solid organ transplantation.
- Orphan drug designation by FDA for the prevention of antibody-mediated organ rejection in solid organ transplant patients
- Orphan drug designation by FDA for the treatment of Guillain Barré syndrome
- Orphan drug designation by FDA for the treatment of anti-GBM antibody disease.
In addition, the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has issued a positive opinion on Orphan Drug Designation for imlifidase for the treatment of anti-glomerular basement membrane (anti-GBM) antibody disease. A positive opinion by the COMP precedes official designation by the European Commission.