Drugs

BridgeBio Plans NDA for Muscular Dystrophy Therapy

BridgeBio Pharma plans to file an NDA next year after reporting positive data from Phase 3 FORTIFY trial evaluating BBP-418, an oral small-molecule therapy for limb-girdle muscular dystrophy type 2I/R9. The progressive genetic muscular dystrophy is caused by FKRP mutations, which impair glycosylation of alpha-dystroglycan (αDG) and lead to loss of mobility, respiratory decline, and cardiac complications, according to the company. There are currently no FDA-approved therapies that modify the disease.

BridgeBio says the pivotal data showed:

Restoration of αDG glycosylation: 1.8-fold improvement from baseline at 3 months, sustained at 12 months
Muscle damage marker impact: 82% mean reduction in serum creatine kinase at 12 months
Motor function improvement: 0.27 meters per second faster velocity versus placebo on the 100-meter timed test
• Pulmonary function gains: Approximately 5% difference vs placebo in forced vital capacity at 12 months

Safety was described as well-tolerated, with no new concerns relative to prior clinical experience.

The company says it will meet with FDA later this year to finalize its regulatory pathway. BBP-418 holds orphan drug, fast track, and rare pediatric disease designations in the U.S., positioning it for potential priority review voucher eligibility if approved.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome