The U.S. Food and Drug Administration published briefing documents regarding upcoming advisory committee meetings for Capricor Therapeutics’ Duchenne muscular dystrophy therapy and Replimune’s advanced melanoma drug, according to a July 29 report. The agency indicated that approvals are uncertain for both therapies.
The FDA disagrees with Capricor’s claim that its cell therapy met the primary endpoints in a Phase 3 trial. For Replimune, the agency continues to have concerns about the use of a single-arm study to support its candidate for advanced melanoma.
Some regulatory experts say they are surprised that the FDA has resumed advisory committee meetings while still increasing staff levels, following a period when such meetings were largely paused under former Commissioner Marty Makary. Others question the rationale behind holding these particular meetings.
In business developments, GSK’s stock increased after unveiling a $2.5 billion cost-cutting plan, while AstraZeneca CEO Pascal Soriot said Monday that the company is “very much on track” to reach an $80 billion revenue target by 2030 despite recent setbacks in clinical trials. Additional second quarter earnings reports from Sanofi, Bristol Myers Squibb, Moderna and others are expected this week. Several smaller biotech companies are also beginning to report their results.
Sarepta announced Michael Severino as its new leader on Tuesday. Severino previously served as CEO of Tessera at Flagship Pioneering and now leads Sarepta as it seeks full FDA approval for exon skipping drugs Amondys 45 and Vyondys 53 after both failed confirmatory trials.
Amgen has launched its official defense of Tavneos, following an FDA recommendation for withdrawal of the rare disease drug.
