Centessa Pharmaceuticals gets FDA orphan drug status for SerpinPC in hemophilia B


Centessa Pharmaceuticals has secured orphan drug designation from the US Food and Drug Administration (FDA) for SerpinPC in hemophilia B treatment.

According to the UK-based clinical-stage pharma company, SerpinPC is an inhibitor of activated protein C (APC).

Centessa Pharmaceuticals is looking at Q4 2022 to kick-start registrational studies of SerpinPC.

Based on the serpin family of proteins, SerpinPC can rebalance coagulation in hemophilia patients by triggering an increase in the production of thrombin by inhibiting activated protein C, said Centessa Pharmaceuticals.

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Last year, around this time, the British pharma company released positive topline data from a phase 2a study of SerpinPC in patients having severe hemophilia A and B, who are not on prophylaxis.

In the highest dose cohort, the APC inhibitor showed a reduction of 88% in median annualized bleeding rate (ABR) for all bleeds, while decreasing median ABR for spontaneous joint bleeds by 94%.

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SerpinPC was well-tolerated without any thrombosis and instances of sustained elevations in D-dimer during the phase 2 a study, said Centessa Pharmaceuticals.

Saurabh Saha — Centessa Pharmaceuticals CEO said: “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.

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“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.

“We look forward to initiating registrational studies for SerpinPC later this year, as well as reporting the two-year follow-up data from the SerpinPC Phase 2a open label extension study in the fourth quarter.”

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