The final minutes from uniQure’s pre–biologics license application (BLA) meeting with the U.S. Food and Drug Administration (FDA) confirm that the company faces a more difficult regulatory path than expected for its Huntington’s disease gene therapy, AMT-130.
According to uniQure’s statement, the FDA indicated that data from Phase I/II studies of AMT-130 are currently unlikely to provide sufficient primary evidence to support a BLA submission. The company is reviewing this feedback and plans to seek another meeting with the agency in early 2026.
UniQure first reported highly positive three-year data from its pivotal trial in September, noting that AMT-130 slowed disease progression by 75%. At that time, shares surged as the company planned to file for approval in early 2026. However, following an initial update on November 3 where uniQure disclosed that the FDA had changed its position regarding the adequacy of current trial data, shares dropped sharply—from $67.49 at close before the announcement to just over $26, and further down to under $23 as of December 4.
Stifel analysts noted Thursday morning that investors’ hopes for a rapid reversal by regulators have diminished: “As time has passed since the 11/3 announcement, we think investors’ optimism has waned around a quick reversal at FDA.” They added there are still several possible outcomes ranging from requiring new studies, following more patients for longer periods, or even a potential reversal by the FDA influenced by external factors.
William Blair analysts expressed surprise at the shift in guidance given ongoing interactions between uniQure and the FDA since November 2024. They pointed out that uniQure’s trial design aligns with recent agency publications outlining mechanisms for personalized therapies and draft guidance suggesting external controls may be acceptable in certain situations.
The regulatory environment has become less predictable for biotech firms this year. Since July, multiple companies have had previous agreements with regulators reconsidered or reversed regarding what evidence is needed for drug approval—a trend some experts believe signals stricter oversight going forward.
If ultimately approved, AMT-130 would represent the first genetic treatment option available for Huntington’s disease.
