BridgeBio eyes first FDA approval for limb-girdle muscular dystrophy therapy

BridgeBio Pharma is preparing for a potential launch of its experimental drug BBP-418 aimed at treating limb-girdle muscular dystrophy type 2I/R9, pending FDA decision.

The agency has set a review deadline for November 27 and has granted priority review without planning an advisory committee meeting, which the company sees as a favorable sign.

Company officials say the trial data showed meaningful improvements in walking speed and respiratory function, addressing a disease that often leads to loss of ambulation and heart or lung complications.

If the drug receives approval, it would become the first disease-modifying treatment for this rare neuromuscular disorder, offering a new option for patients who currently have limited therapies.