Business
Krystal Biotech Receives FDA Approval for the First-Ever Redosable Gene Therapy, VYJUVEK™ (beremagene geperpavec-svdt) for the Treatment of Dystrophic Epidermolysis Bullosa
Dystrophic Epidermolysis Bullosa (DEB) is a serious rare genetic disease that affects the skin and mucosal tissues and is caused by one or more mutations in
About this update from Krystal Biotech, Inc.
[{"type":"text","content":"Dystrophic Epidermolysis Bullosa (DEB) is a serious rare genetic disease that affects the skin and mucosal tissues and is caused by one or more mutations in the COL7A1 gene, resulting in lack of production of functional type VII collagen (COL7) proteinVYJUVEK is a topical gel that addresses the genetic cause of DEB by restoring functional copies of the COL7A1 gene to patients and is the only medicine available for patients in the USVYJUVEK is approved for the treatment of patients six months of age or older with either recessive or dominant DEBVYJUVEK is approved to be administered by a healthcare professional either in a healthcare professional setting (e.g., clinic) or a home settingData from the pivotal Phase 3 (GEM-3) trial, published in the New England Journal of Medicine, demonstrated that the trial met its primary endpoint of complete wound healing at six months and its key secondary endpoint of complete wound healing at three monthsRare Pediatric Disease Priority Review Voucher grantedCompany to host investor conference call on Friday, May 19, 2023 at 6:00 pm ET PITTSBURGH, May 19, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the Company) (NASDAQ: KRYS), a biotechnology company focused on developing and commercializing genetic medicines for patients with rare diseases, today announced the US Food and Drug Administration (FDA) has approved VYJUVEK™ (beremagene geperpavec-svdt) for the treatment of patients six months of age or older with dystrophic epidermolysis bullosa (DEB). VYJUVEK is designed to address the genetic root cause of DEB by delivering functional copies of the human COL7A1 gene to provide wound healing and sustained functional COL7 protein expression with redosing. VYJUVEK is the first-ever redosable gene therapy and the first and only medicine approved by the FDA for the treatment of DEB, both recessive and dominant, that can be administered by a healthcare professional in either a healthcare professional setting or in the home. DEB is a rare and serious disease that affects the skin and mucosal tissues caused by one or more mutations in the COL7A1 gene. The COL7A1 gene is responsible for the production of functional COL7 protein that forms anchoring fibrils necessary to bind the dermis (inner layer of the skin) to the epidermis (outer layer of the skin). The lack of functional anchoring fibrils in DEB ...