- The FDA grants Orphan Drug status for Omeros' (OMER +8.9%) lead candidate OMS721 for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), a severe complication of HSCT characterized by blood clotting in the smallest blood vessels in the body.
- Among the benefits of Orphan Drug status is a seven-year period of market exclusivity, if approved.
- The FDA designated OMS721 a Breakthrough Therapy for the indication in April.
- OMS721 binds to an enzyme called mannan-binding lectin-associated serine protease-2 (MASP-2) that plays a key role in the lectin pathway of the complement system which is believed to contribute to significant tissue injury.
- A U.S. marketing application is in process.
- Previously: FDA designates Omeros' OMS721 for accelerated review for stem cell transplant-associated clotting disorder; shares up 9% (April 26)
- Previously: Omeros up 23% on preparations for BLA for OMS721 (May 11)
- Now read: Your Daily Pharma Scoop: Intra-Cellular Therapies Completes Rolling NDA, Amarin Rallies Further, Insmed's Approval
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