The Company has filed the Marketing Authorization (MA) application with the European Medical Agency (EMA) and is currently working closely with the EMA through the MA validation process.Ībout Dystrophic Epidermolysis Bullosa (DEB)ĭEB is a rare and severe disease that affects the skin and mucosal tissues. The BLA was granted Priority Review designation and the Prescription Drug User Fee Act action date is February 17, 2023. The Company received US Food and Drug Administration (FDA) filing acceptance of its Biologics License Application (BLA) for B-VEC. “B-VEC was developed by Krystal scientists as a potential first-in-class therapy for DEB,” said Suma Krishnan, President, Research & Development, Krystal Biotech. “We are working closely with the FDA to get B-VEC approved and deliver a meaningful benefit to patients with this debilitating disease.” B-VEC was well tolerated, with no drug-related serious adverse events or discontinuations due to treatment. The pivotal GEM-3 trial met its primary endpoint of complete wound healing at six-months and its secondary endpoint of complete wound healing at three-months. In the trial, matched wounds receiving topical B-VEC or placebo were evaluated in 31 DEB patients over 26 weeks. The GEM-3 trial was a randomized, double-blind, intra-patient placebo-controlled multi-center trial designed to evaluate the efficacy and safety of B-VEC for the treatment of DEB. “The impressive phase 3 results with B-VEC are the best we have seen to date in patients with DEB and, if approved, B-VEC provides hope for these patients suffering through debilitating and potentially life-threatening symptoms associated with the disease, ” said Peter Marinkovich, M.D., Director of the Blistering Disease Clinic at Stanford Health Care, Associate Professor of Dermatology at the Stanford University School of Medicine, primary investigator of the GEM-3 trial and primary author of the manuscript. Complete wound healing at 3 months occurred in 70.6% of the wounds exposed to B-VEC as compared with 19.7% of those exposed to placebo (difference, 51.0 percentage points 95% CI, 29.3 to 72.6 p=0.0005). In this GEM-3 trial of 31 patients, complete wound healing at 6 months occurred in 67.4% of B-VEC wounds compared to 21.6% for placebo (difference, 45.8 percentage points 95% confidence interval, 23.6 to 68.0 p=0.002). The full manuscript, titled “Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa,” will appear in the December 15, 2022 issue of the NEJM. (the “Company”) (NASDAQ: KRYS), a biotechnology company focused on developing and commercializing genetic medicines for patients with rare diseases, announced today that data from the pivotal Phase 3 (GEM-3) trial of beremagene geperpavec (B-VEC) for dystrophic epidermolysis bullosa (DEB) have been published here in the New England Journal of Medicine (NEJM). 14, 2022 (GLOBE NEWSWIRE) - Krystal Biotech, Inc.
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