Rare Daily Staff

The U.S. Food and Drug Administration granted BridgeBio Pharma Fast Track designation to its experimental gene therapy designed for the treatment of congenital adrenal hyperplasia, a rare hormonal condition.

The FDA grants Fast Track designation 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. The gene therapy, known as 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).

CAH is one of the most prevalent genetic diseases with more than 75,000 cases estimated in the United States and Europe. The 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.

BBP-631 is designed to provide a functional copy of the 21-hydroxylase-encoding gene to help patients produce their own cortisol and aldosterone.

“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,” said Eric David, CEO at BridgeBio Gene Therapy. “The FDA’s Fast Track designation reinforces the urgency to address the unmet needs of patients with CAH as quickly and safely as possible.”

The FDA has cleared the company’s Investigational New Drug application and site activation for the phase 1/2 study is ongoing and is expected to begin in the coming months.

Photo: Eric David, CEO at BridgeBio Gene Therapy

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