Drugs

Sarepta Completes Confirmatory Duchenne Trial

Sarepta Therapeutics says its long-running Phase 3 ESSENCE confirmatory study evaluating its Duchenne muscular dystrophy drugs Amondys 45 (casimersen) and Vyondys 53 (golodirsen) showed mixed data, but the company still plans to meet with FDA to discuss converting the therapies from accelerated to traditional approval.

The nine-year, placebo-controlled trial enrolled 225 boys ages 6 to 13 with Duchenne amenable to exon 45 or 53 skipping. Sarepta says the study showed “numerical trends” favoring treatment over placebo. Still, the results did not reach statistical significance on the primary endpoint — improvement in four-step ascend velocity at 96 weeks.

Sarepta says that when excluding data from 57 participants whose blinded treatment period overlapped with the Covid-19 pandemic, treated patients showed a 30% reduction in disease progression (0.11 steps per second), a change it described as clinically meaningful.

No new safety concerns emerged, Sarepta reports, adding that most adverse events were mild or moderate, and the safety profile was consistent with previous data from the company’s exon-skipping therapies. Common side effects included vomiting, nasopharyngitis, fever, headache, cough, and upper respiratory infections.

“While the ESSENCE study did not meet statistical significance on its primary endpoint, we believe the results demonstrated a clear treatment effect,” a company release says. “These findings reinforce the potential impact of these therapies to slow muscle weakness and other symptoms.”

Sarepta says the ESSENCE data, together with extensive real-world evidence, will be submitted to FDA as part of a planned supplemental NDA for both drugs, adding that the study’s completion fulfills their primary postmarketing requirements.

Both exon-skipping therapies were previously granted accelerated approval based on increases in dystrophin production, a protein lacking in patients with Duchenne, according to the company.

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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