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.

In January 2017, EMA approved Hansa Medical’s application for orphan drug designation of IdeS for the prevention of graft rejection following solid organ transplantation. In September 2015, IdeS was granted orphan drug designation for the prevention of antibody-mediated organ rejection in solid organ transplant patients by the FDA.

In February 2018, the FDA granted orphan drug designation to IdeS (INN: imlifidase) for the treatment of GBS.