CAH is caused by a deficiency in the 21-hydroxylase enzyme, which is critical for hormone production necessary for physiological functions.

If approved, BBP-812 could become the first therapeutic option for children suffering from Canavan disease, a severe neurodevelopmental disorder with significant unmet medical needs.

The CANaspire trial, which evaluated BBP-812, demonstrated promising results in safety and efficacy, focusing on the reduction of N-acetylaspartate (NAA) levels in pediatric patients.

The RMAT designation for BBP-812 allows for early and frequent interactions with the FDA, potentially facilitating an Accelerated Approval pathway.

BridgeBio's strategic decision to allocate resources more effectively may enhance its overall financial health as it focuses on priority targets.

BridgeBio Pharma has announced the discontinuation of its development of BBP-631, a gene therapy aimed at treating Congenital Adrenal Hyperplasia (CAH).

Despite showing some efficacy, including substantial increases in 11-deoxycortisol and significant reductions in 17-hydroxyprogesterone, the results from the Phase 1/2 study of BBP-631 were not sufficient to justify further investment.

BBP-631 was generally well tolerated, with only mild to moderate treatment-emergent adverse events reported and no serious adverse events linked to the therapy.

BridgeBio is now seeking partnership opportunities to support the future development of BBP-631 or next-generation gene therapies for CAH, which affects approximately 75,000 people in the U.S. and EU.

In a positive development, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to BridgeBio's investigational gene therapy BBP-812, aimed at treating Canavan disease.

Founded in 2015, BridgeBio Pharma is dedicated to developing transformative medicines for genetic diseases, aiming to expedite patient access to innovative therapies.

The therapy achieved a maximum cortisol level of 11 µg/dL, which fell short of the threshold set by CEO Neil Kumar for advancing the treatment.