Complete response letters
After devastating regulatory setbacks last year, shares of uniQure and Replimune rose on the news that FDA Commissioner Marty Makary is leaving. But analysts don’t expect much relief, no matter who helms the agency.
The FDA’s decision last year to make complete response letters public provides new insight into why therapies sometimes fail to get the regulatory greenlight. Analysts say the information could help sponsors refine their regulatory strategies.
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.
Some 30% of clinical trials that are mandated to report their findings have not posted results to clinicaltrials.gov, the federal government’s public repository for studies, according to internal data from the FDA.
Replimune’s CEO Sushil Patel has already warned that the biotech will need to cut staff and substantially scale back its U.S. manufacturing operations.
The FDA in a complete response letter to Replimune maintained its original objection to the single-arm trial the biotech used to support the application for RP1.
The upcoming FDA decision for Replimune’s advanced melanoma drug could be a litmus test for the agency’s future regulatory decision-making, analysts say, with implications stretching well beyond one company.
The U.S. Senate has a plan to improve drug development for rare disease patients. The exit of controversial CBER chief Vinay Prasad will help clear the path.
Industry and FDA representatives have reached a general agreement on planned pre-submission facility meetings but have expressed different views about the specifics.
Incyte is looking to expand the use of its PD-1 blocker Zynyz to treat non-small cell lung cancer. The FDA’s rejection did not flag problems with the drug’s efficacy or safety.
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