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BridgeBio's genetic muscle disorder drug meets all goals in late-stage trial
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BridgeBio's genetic muscle disorder drug meets all goals in late-stage trial
Oct 27, 2025 4:18 AM

Oct 27 (Reuters) - BridgeBio Pharma ( BBIO ) 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.

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