Rare diseases
After an FDA advisory committee voted 9-3 on Wednesday against recommending Capricor Therapeutics’ Duchenne muscular dystrophy cardiomyopathy drug for approval, stakeholders allege an “ulterior motive” and “biased intentions.”
Alnylam’s revenue cut “may create a near term credibility overhang and could “raise questions on peak sales potential, even if there’s tailwinds in the competitive landscape,” according to Stifel.
UniQure is ready to put its Huntington’s disease gene therapy through an advisory committee meeting—even as the broader rare disease space has been put on edge by recent FDA scrutiny of assets by Replimune and Capricor.
During a perplexing advisory committee meeting in which FDA reviewers focused largely on the Phase 3 HOPE-3 trial’s secondary endpoint of cardiac function, experts pointed to “very fragile” data. The committee voted 9-3 against approval of deramiocel.
The FDA published briefing documents ahead of advisory committee meetings for therapies from Capricor Therapeutics and Replimune indicate an uphill battle for the biotechs; GSK and AstraZeneca outline ambitious plans in second quarter presentations; Sarepta taps Michael Severino as its new CEO; and Amgen launches its official defense of Tavneos.
Oak Hill Bio is developing the antisense therapy rugonersen, which it licensed from Roche in 2025, for Angelman syndrome.
In briefing documents published Monday ahead of an advisory committee meeting for deramiocel, the FDA disagreed with Capricor’s assertion that a Phase 3 trial met its primary outcome measures.
After leaving Tessera Therapeutics earlier this month, Michael Severino will take over Sarepta Therapeutics as the biotech awaits two key regulatory decisions in Duchenne muscular dystrophy.
Facing pressure around the world to withdraw the rare kidney disease drug Tavneos, Amgen has launched a robust defense with real-world evidence to support the product’s benefit-risk profile.
With Phase 3 data showing what analysts called a “potentially best-in-class profile,” Arrowhead Pharmaceuticals plans to submit for FDA approval of plozasiran by the end of the year—hoping to follow to market Ionis Pharmaceuticals’ Tryngolza, which got the regulator’s nod in severe hypertriglyceridemia last month.
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