Drugs

BridgeBio Data on BBP-418 in Limb-Girdle Muscular Dystrophy

BridgeBio Pharma says its investigational therapy BBP-418 showed consistent clinical benefit and a favorable safety profile in an interim analysis of the Phase 3 FORTIFY trial in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), a rare and progressive genetic muscle disorder with no approved treatments. LGMD2I/R9 is caused by mutations in the FKRP gene and leads to progressive muscle weakness, loss of ambulation and other complications. Currently, there are no disease-specific therapies for the condition.

Based on the interim results, BridgeBio says it plans to submit an NDA in the first half of 2026 seeking traditional approval of BBP-418.

The interim analysis showed early separation from placebo on the 100-meter timed test, a measure of ambulation, with differences emerging as early as three months after treatment initiation. After 12 months, patients receiving BBP-418 completed the test about 31 seconds faster than those on placebo. Participants treated with BBP-418 also showed improvement in the 10-meter walk test, with walking speed increasing by 0.13 meters per second from baseline, while patients on placebo experienced a decline of 0.10 m/s.

The company says the therapy also produced rapid and sustained reductions in serum creatine kinase (CK), a biomarker associated with muscle damage. Nearly 60% of treated patients had CK levels reduced to within twice the upper limit of normal after one year, and 38% achieved normalization. Treatment effects were consistent across prespecified patient subgroups, including differences in genotype, age and baseline lung function.

Investigators reported that BBP-418 was generally well tolerated, according to the company. Treatment-emergent adverse events occurred in 93.2% of patients receiving the therapy and 100% of those receiving placebo, while severe adverse events occurred at similar rates in both groups. The most common side effects included diarrhea, procedural pain, falls, upper respiratory infections, nausea and joint pain. All cases of diarrhea were mild to moderate, the company says.

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

Read more

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