BridgeBio Pharma Receives FDA Fast Track Designation for Investigational Gene Therapy for Congenital Adrenal Hyperplasia

BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, today announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to BBP-631, an investigational adeno-associated virus 5 (AAV5) gene therapy designed for the treatment of congenital adrenal hyperplasia (CAH).

Fast Track designation, granted by the FDA, is designed to facilitate the development and to expedite the review of new therapies hoping to treat or prevent serious conditions and fill an unmet medical need. Additionally, BBP-631 was granted Rare Pediatric Disease Designation by the FDA and has received Orphan Drug Designation by the FDA and European Medicines Agency (EMA).

“The standard-of-care for CAH patients has not changed significantly over the last 50 years, and a gene therapy offers for the first time the possibility that patients may be able to make their own cortisol and aldosterone, at the right times and in the right amounts. The FDA’s Fast Track designation reinforces the urgency to address the unmet needs of patients with CAH as quickly and safely as possible. We are grateful to have received Fast Track along with other key designations granted by the FDA and the EMA,” said Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy, which is focused on developing gene therapy treatment options for patients in need. “We are eager to initiate our first-in-human Phase 1/2 study. The Investigational New Drug application has been cleared by the FDA and site activation for the study is ongoing, and is expected to begin in the coming months.”

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