Benzinga | Sep 28, 2021 04:02PM EDT

FDA Grants Catalyst Biosciences Orphan Drug Designation For MarzAA For The Treatment Of Factor VII Deficiency

Catalyst Biosciences, Inc. (NASDAQ:CBIO) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Disease Designation (ODD) for its lead product candidate, subcutaneous Marzeptacog alfa (activated), or MarzAA, for the treatment of Factor VII Deficiency (FVIID). MarzAA was previously granted ODD and Fast Track Designation (FTD) for treatment of Hemophilia A/B with inhibitors and FTD for the treatment of FVIID.



"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, Ph.D., president and chief executive officer of Catalyst.

The FDA grants Orphan Drug Designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides benefits to drug developers designed to support the developments of drugs and biologics for small patient populations with unmet medical needs. These benefits include assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.