- The U.S. Food and Drug Administration (FDA) granted orphan drug designation to Centessa Pharmaceuticals' ( NASDAQ: CNTA ) SerpinPC to treat hemophilia B.
- The U.K.-based company said in a Sept. 14 press release that it plans to begin registrational studies of SerpinPC in Q4 2022.
- SerpinPC is a biological drug candidate, based on the serpin family of proteins which is designed to allow more thrombin to be generated by inhibiting activated protein C (APC), thus rebalancing coagulation in patients with hemophilia, according to Centessa.
- "We believe SerpinPC has the potential to offer patients with hemophilia B a convenient subcutaneous option that is designed to prevent and reduce bleeds without the risk of thrombosis," said Centessa CEO Saurabh Saha.
- The FDA grants orphan drug status to therapies which treat or prevent rare diseases that affect fewer than 200K people in the U.S. The designation provides certain incentives, including up to seven years of market exclusivity, if approved.
For further details see:
Centessa hemophilia B therapy SerpinPC gets FDA orphan drug status