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Krystal Biotech Submits Biologics License Application to U.S. FDA Seeking Approval of B-VEC for the Treatment of Patients with Dystrophic Epidermolysis Bullosa
PITTSBURGH, June 22, 2022 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), the leader in redosable gene therapy, announced today the
About this update from Krystal Biotech, Inc.
[{"type":"text","content":"PITTSBURGH, June 22, 2022 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), the leader in redosable gene therapy, announced today the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval of B-VEC (beremagene geperpavec) for the treatment of patients with dystrophic epidermolysis bullosa (DEB). B-VEC is an investigational non-invasive, topical gene therapy designed to treat DEB at the molecular level by providing the patient’s skin cells with two copies of the COL7A1 gene to make functional COL7 protein, thereby addressing the fundamental disease-causing mechanism. “The unmet medical need for DEB patients remains very high and our relentless pursuit of a treatment for this disease continues with the same sense of urgency that we have always had since the founding of Krystal Biotech,” said Suma Krishnan, President of Research & Development. “We look forward to working with the FDA in its review of our BLA submission.” The BLA submission for B-VEC is supported by data from two placebo controlled clinical trials - the GEM-3 trial (NCT04491604) and the GEM-1/2 trial (NCT03536143). 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. In the trial, matched wounds receiving topical B-VEC or placebo were evaluated in 31 DEB patients over 26 weeks. 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. B-VEC was well tolerated, with no drug-related serious adverse events or discontinuations due to treatment. The GEM-1/2 trial was a randomized, open-label, intra-patient placebo-controlled single-center trial designed to evaluate efficacy (mechanistic and clinical) and safety of B-VEC for the treatment of DEB. In the trial, matched wounds receiving topical B-VEC or placebo were evaluated in nine recessive dystrophic epidermolysis bullosa patients over 12 weeks. Both mechanistic and clinical endpoints were met. No serious or severe B-VEC-related adverse events or systemic drug exposure were noted. Results from the GEM-1/2 trial of B-VEC for the treatment of DEB were published in Nature Medicine. The publication provides a compreh...