FDA Grants Orphan Drug Status To Centessa's Hemophilia B Candidate

Centessa Pharmaceuticals CNTA announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to SerpinPC for the treatment of hemophilia B.

A Phase 2a proof-of-concept study evaluated SerpinPC in severe hemophilia A and B patients not on prophylaxis.

SerpinPC is a biological drug candidate, based on the serpin family of proteins that is designed to allow more thrombin to be generated by inhibiting APC, accordingly rebalancing coagulation in hemophilia patients.

The company reported positive top-line data from the study, SerpinPC was observed to be well-tolerated with no thrombosis and no instances of sustained D-dimer elevations.

Saurabh Saha, Chief Executive Officer, stated, "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. This designation from the FDA is an important milestone in the development of SerpinPC and underscores the need for new, innovative treatment options for patients with hemophilia B.”

The company anticipates initiating registrational studies for SerpinPC later this year, and planning to report two-year follow-up data from the Phase 2a open label extension study in the fourth quarter.

FDA grants orphan designation to promote the development of a drug that is expected to have significant therapeutic advantage over existing treatments that target a condition affecting 200,000 or fewer U.S. patients annually.

Price action : Centessa shares are trading around 4 percent down at $4.09 on Wednesday at the time of publication.