The FDA has changed its position on two separate therapies in recent weeks, allowing a melanoma treatment and a Huntington’s disease gene therapy to advance toward approval after earlier rejections.
Replimune reached an agreement with the FDA on a path forward for its approval application for RP1 (vusolimogene oderparepvec), an experimental cancer immunotherapy for advanced melanoma, according to a May 29 news release. The company will resubmit the application in the coming days, and the FDA indicated it will treat it as an urgent matter and prioritize its review in recognition of significant unmet need.
The FDA had issued a complete response letter for RP1 in April — the second rejection of the therapy. The drug was first rejected in July 2025, with the FDA raising concerns about Replimune’s reliance on a single-arm trial to support the application.
On the Huntington’s front, uniQure announced that the FDA agreed three-year data from the company’s Phase 1/2 study would be acceptable as the primary basis for an approval application for AMT-130, with a submission planned for the third quarter of 2026, according to a June 17 news release.
The reversal marks a significant shift. In November 2025, uniQure said the FDA “no longer agrees” that the company’s trial data would be adequate to support an approval application — what the company called “a drastic change” from guidance the FDA had provided just a year earlier. The FDA had previously granted AMT-130 both Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations.
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