FDA
ITM Isotope Technologies plans on resubmitting its advanced cancer radiopharma therapy to the FDA after addressing the agency’s concerns.
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The FDA has been trying to mend fences with rare disease drug developers and patient groups since the departure of former Commissioner Marty Makary three months ago. But advisory committee meetings last week for Replimune and Capricor Therapeutics only created more confusion and frustration.
The FDA’s new investigational new drug pilot program—one part of HHS’s broader clinical trial modernization initiatives—has lofty goals to expedite first-in-human trials of novel drugs, but experts say it won’t tip the scales much on its own.
The FDA will hold advisory committee meetings this week for previously rejected investigational therapies from Replimune and Capricor Therapeutics. Some industry leaders were surprised the meetings were called at all, while many have questioned the rationale behind them and other adcomms on deck.
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The industry has been abuzz since the weekend when talks of a potential mega-merger between Bristol Myers Squibb and AstraZeneca were reported. Meanwhile, Pfizer, Merck and others release second quarter earnings, with some disclosing pipeline discontinuations and cost-cutting measures. Finally, all eyes turn to the FDA in the wake of heated advisory committee meetings for Capricor Therapeutics and Replimune.
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.”
Moderna’s norovirus vaccine failed in a Phase 3 trial, but the company still has faith in an ambitious revenue growth target thanks to ex-U.S. vaccine sales and a unanimous FDA adcomm win for mFLUSIVA.
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.
The FDA’s Cellular, Tissue and Gene Therapies advisory committee voted 10 to 3 in favor of Replimune’s immunotherapy for advanced melanoma in combination with Bristol Myers Squibb’s Opdivo, but trial design was a major sticking point as the panel deliberated.
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.
The FDA’s rebuff of Replimune comes a day after the agency similarly called into question the Phase 3 trial for Capricor’s Duchenne muscular dystrophy cell therapy.
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.
Despite a Phase 3 miss, Gossamer Bio is full steam ahead for its lung disease asset, pointing to previous “review issues” at the FDA as one of the reasons to continue development.