BridgeBio Gets FDA Fast Track Designation For Gene Therapy For Congenital Adrenal Hyperplasia

BridgeBio Pharma Inc. (BBIO) said that the U.S. Food and Drug Administration granted Fast Track designation to BBP-631, an investigational adeno-associated virus 5 gene therapy designed for the treatment of congenital adrenal hyperplasia.

Fast Track designation 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.

In addition, BBP-631 was granted Rare Pediatric Disease Designation by the FDA and has received Orphan Drug Designation by the FDA and European Medicines Agency.

The congenital adrenal hyperplasia disease is caused by deleterious mutations in the gene encoding an enzyme called 21-hydroxylase, leading to lack of endogenous cortisol and aldosterone production. This lack of production causes patients with CAH to be unable to form physiological responses to illnesses and stressors, which can be life-threatening, especially for children.

The Investigational New Drug application has been cleared by the FDA and site activation for initiation of a first-in-human Phase 1/2 study is ongoing, with initial data anticipated in late 2021 or early 2022.

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