(Reuters) -BridgeBio Pharma said on Monday its experimental drug for a rare muscle disorder, which currently has no approved treatments, showed improvements in motor and lung function in a late-stage trial.
The oral drug, BBP-418, is being tested in patients with a form of limb-girdle muscular dystrophy, or LGMD, a group of inherited genetic disorders characterized by progressive muscle weakness and wasting.
LGMD has an estimated global prevalence ranging from 1 to 6 per 100,000 people, with about 5,000 people in the U.S. currently living with the condition.
BBP-418 met the main goal of significantly increasing glycosylated αDG, a key marker of LGMD, by 1.8 times from baseline when compared to placebo in an interim analysis of the trial at three months.
Glycosylated αDG is a form of the protein αDG that has undergone proper glycosylation, a biochemical modification essential for its function in muscle cells.
The therapy at 12 months showed a statistically significant difference in serum CK, a marker of muscle damage, an increase in walking speed by 0.27 metre/second and an improvement of about 5% in the volume of air participants could forcibly exhale when compared to placebo.
BridgeBio intends to engage with the U.S. Food and Drug Administration later this year to discuss these data and plans to submit a marketing application for BBP-418 in the first half of 2026.
The company plans to present detailed results from the trial at a future medical meeting.
(Reporting by Christy Santhosh in Bengaluru; Editing by Shreya Biswas)
Comments