BridgeBio’s Experimental Drug Hits Key Goals in Major Muscle-Disorder Trial

BridgeBio Pharma announced that its experimental medication for a rare genetic muscle disorder successfully met all primary and secondary targets in a late-stage study.

The drug, aimed at treating Limb‑Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), a progressive condition affecting the muscles close to the hips and shoulders, demonstrated significant improvements in muscle markers compared to placebo.

Company executives said the trial’s results support a potential regulatory filing, with plans to submit an approval application soon. They added this therapy could become the first approved treatment for LGMD2I/R9.

Investors responded positively to the news, sending BridgeBio’s shares higher and improving confidence in its pipeline of genetic-disease treatments. Analysts cited this as a major milestone for an area of high unmet need.

Still, experts noted the therapy must now pass regulatory review and prove its benefits in longer-term real-world use before full approval and commercial marketing.