FDA Grants MarzAA Orphan Drug Designation

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FDA granted orphan drug designation to Catalyst Biosciences’ MarzAA for the treatment of Factor VII Deficiency.

Catalyst Biosciences, a research and clinical development biopharmaceutical company, announced on Sept. 28, 2021, that FDA has granted orphan drug designation (ODD) to its lead product candidate, subcutaneous Marzeptacog alfa (activated), or MarzAA, for the treatment of Factor VII Deficiency (FVIID).

MarzAA is a subcutaneously administered next-generation engineered coagulation Factor VII designed for the treatment of episodic bleeding in subjects with rare bleeding disorders. FVIID is a rare condition that interferes with the blood clotting process, resulting in prolonged bleeding.

MarzAA had previously been granted ODD and fast track designation (FTD) for the treatment of another blood disorder, Hemophilia A/B, with inhibitors. Additionally, MarzAA was granted FTD for FVIID treatment by FDA prior to this ODD.


"Receiving a second orphan drug designation in addition to two FTDs for MarzAA demonstrates its potential in treating multiple rare bleeding disorders” said Nassim Usman, president and CEO of Catalyst Biosciences, in a company press release.

Source: Catalyst Biosciences