CBIO - Catalyst granted FDA's orphan drug designation for hemophilia drug

The FDA has granted the orphan drug designation to Catalyst Biosciences (NASDAQ:CBIO) for its lead candidate marzeptacog alfa as a treatment for bleeding in patients with hemophilia A or B. With its orphan drug designation, the federal agency aims to offer financial incentives to drug developers targeting rare diseases and conditions. In addition to tax credits for clinical trial costs and waiver of the user fee for marketing applications, the developers of orphan drugs can claim seven years of marketing exclusivity upon regulatory approval for the treatment. In 2019, Catalyst (CBIO) shares soared in reaction to Orphan Drug status granted in Europe for marzeptacog alfa, a Factor VIIa variant also known as MarzAA. In September, the FDA issued Orphan Drug Disease Designation for subcutaneous marzeptacog alfa as a treatment for Factor VII Deficiency.

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Catalyst granted FDA’s orphan drug designation for hemophilia drug