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FDA Grants Rare Pediatric Disease Designation to Omeros’ MASP-3 Inhibitor Zaltenibart for Treatment of C3 Glomerulopathy
-- Phase 3 Studies on the Horizon in both PNH and C3G -- SEATTLE--(BUSINESS WIRE)-- Omeros Corporation today announced that zaltenibart (OMS906) has received
About this update from Omeros Corporation
[{"type":"text","content":"\n-- Phase 3 Studies on the Horizon in both PNH and C3G --\n\n SEATTLE--(BUSINESS WIRE)--\n\nOmeros Corporation today announced that zaltenibart (OMS906) has received rare pediatric disease designation from the U.S. Food and Drug Administration (FDA) for the treatment of complement 3 glomerulopathy (C3G), an ultra-rare, progressive renal disorder primarily afflicting children and young adults. Caused by dysregulation of the alternative pathway of complement, there is no approved treatment for C3G, which often leads to end-stage renal disease within 10 years of diagnosis. Zaltenibart is the most proximal inhibitor of the alternative pathway. It blocks mannan-binding lectin-associated serine protease-3 (MASP-3), the key activator of the alternative pathway, stopping the conversion of pro-complement factor D (pro-CFD) to mature CFD. Phase 3 clinical trials for zaltenibart in C3G are slated to begin next year.\n\n“C3G is devastating for children as well as for adults, and our receipt of FDA’s rare pediatric disease designation is a welcome acknowledgment of zaltenibart as a potential therapeutic for this disease that has no approved treatment,” stated Gregory A Demopulos, chairman and CEO of Omeros. “With zaltenibart clinical studies ongoing in both PNH and C3G and preparations underway to begin Phase 3 trials, we look forward to bringing zaltenibart to market, expanding its list of targeted indications and demonstrating its advantages over other alternative pathway inhibitors.”\n\nCompanies awarded a rare pediatric disease designation receive a rare pediatric disease priority review voucher from FDA when the designated drug is approved for the associated indication in the pediatric population. The voucher allows the recipient company to obtain FDA priority review of either a New Drug Application (NDA) or Biologics License Application (BLA) for a different product and/or indication, reducing the review time and accelerating any granted approval and subsequent market entry by at least four months. The voucher may be used by the original recipient, or it can be sold to another company for the purchaser’s use.\n\nOmeros is also advancing zaltenibart for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare and life-threatening blood disease. Omeros has received orphan drug designation from FDA for zaltenitbart in th...