FDA
UniQure plans to submit AMT-130 to the U.K.’s Medicines and Healthcare products Regulatory Agency in the third quarter of 2026 based on Phase 1/2 data showing a 75% slowing of disease—the same data the FDA has deemed unacceptable for a biologics license application.
While some analysts consider the return of advisory committees a positive sign for the FDA—and the biopharma industry more widely—others are keeping their optimism in check, waiting instead for more foundational changes at the regulator.
In briefing documents released Wednesday, the FDA raises doubts about two AstraZeneca assets set to be discussed Friday at the agency’s first drug-related advisory committee meeting in nine months.
Sanofi and Novartis kick off the heart of earnings season; Lilly strikes its fourth pact in as many weeks; Regeneron earns landmark approval for a gene therapy for a type of genetic deafness, and also strikes a White House deal; FDA asks Amgen to withdraw Tavneos and, separately, issues Commissioner’s National Priority Vouches to three unnamed psychedelics companies.
While the FDA did not announce the recipient names of the Commissioner’s National Priority Vouchers, the agency’s descriptions of the awarded products match those in development at Compass Pathways, Transcend Therapeutics and Usona Institute.
The FDA is signaling change, but actual success depends on more than simply bringing in a new leader at the Center for Biologics Evaluation and Research; it requires accountability, transparency and consistent action.
The FDA in July 2025 made publicly available over 200 complete response letters—an initiative that the investment community sees as “unanimously positive,” analysts told BioSpace.
The deals keep rolling in, with Lilly penning a $7 billion pact for gene delivery biotech Kelonia Therapeutics and UCB taking over cell therapy-focused Neurona Therapeutics; President Trump signed a new executive order supporting the development of psychedelic therapies, sparking fanfare and concern alike; and the FDA’s recent Replimune decision has triggered broader debate about the agency’s flexibility.
In the U.S., Moderna withdrew its approval application for the combination vaccine in May last year and the timeline for resubmission remains uncertain.
The newly approved HIV drug Idvynso will also help Merck diversify as loss of exclusivity looms over its top-selling product, the mega-blockbuster cancer drug Keytruda.
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