BridgeBio Pharma will seek US approval of its limb-girdle muscular dystrophy (LGMD) small molecule after the drug met its endpoints in a pivotal Phase III trial.
In the FORTIFY study (NCT05775848), ribitol, also known as BBP-418, was associated with improvement in motor and pulmonary function, along with robust restoration of αDG glycosylation in patients with LGMD type 2I/R9 (LGMD2I/R9).
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After BridgeBio announced the data, its stock climbed 17.3% from a 24 October close of $54.26 to a 27 October high of $63.65 (correct at 11am ET 27 October 2025).
In the randomised, double-blind, placebo-controlled study, patients treated with ribitol saw a highly statistically significant 180% change from baseline of glycosylated alpha-dystroglycan (αDG). This is compared to approximately no change in the placebo group at three months. This was sustained throughout the full 12-month study period.
There was also an average 82% reduction in serum creatine kinase (CK), a marker of muscle breakdown in patients who received the study drug.
All key clinical endpoints, including ambulatory function and pulmonary function (FVC), were met with both statistical and clinical significance.
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By GlobalDataFinally, the drug remained well-tolerated with no new or unexpected safety findings observed compared to previous studies.
Dr Katherine Mathews, professor of paediatrics and neurology at the University of Iowa’s Roy J and Lucille A Carver College of Medicine, said: “This is such an important result for individuals living with LGMD2I/R9, which is a progressive muscular dystrophy.
“The resulting weakness often leads loss of ambulation, need for respiratory support, and need for heart failure medications. To date, there has been no specific treatment. These results bring enormous hope that BBP-418 might change the disease course.”
As a result of the Phase III success, BridgeBio will speak with the US Food and Drug Administration (FDA) about plans to submit a new drug application (NDA) for ribitol in LGMD in the first half of 2026.
Ribitol received orphan drug, fast track, and rare paediatric disease designations from the FDA, as well as an orphan drug designation from the European Medicines Agency (EMA).
GlobalData’s annual sales and consensus forecast predict a 2026 approval of BridgeBio’s candidate, with 2031 sales reaching $518m.
GlobalData is the parent company of Clinical Trials Arena.
Barren treatment space
According to GlobalData analysis, there are no approved treatments for LGMD, with most therapies focusing on disease management.
Sarepta was investigating a LGMD candidate, SRP-9004; however, the drug was put under clinical hold by the FDA earlier this year after a patient death was reported. The programme remains paused at this time.
Sarepta will be pinning its hopes on SRP-9003 for LGMD2E/R4, which is in a Phase III trial. At the 30th annual International Congress of the World Muscle Society (WMS), Sarepta announced that the gene therapy led to a 23.9% increase in beta-sarcoglycan expression and a protein expression rise of 43.4%.
