FDA Signals uniQure Huntington Gene Therapy Data Could Back Accelerated Approval

uniQure N.V. (NASDAQ:QURE) stock is up during Wednesday’s premarket session as the company announced plans for a Biologics License Application (BLA) submission for AMT-130, a gene therapy treatment for Huntington’s disease.

FDA Opens Accelerated Approval Path

This news comes following a Type B meeting with the FDA, where the agency indicated that the three-year analysis from the Phase I/II study could serve as the primary basis for accelerated approval. The company intends to submit the BLA in the third quarter of 2026.

uniQure FDA Update On AMT-130 BLA Path

The FDA’s acceptance of the three-year analysis as a basis for the BLA submission marks a significant milestone for uniQure.

Earlier, the FDA advised against using Phase 1/2 study data as the primary evidence for the marketing application of AMT-130, raising uncertainty around the therapy's regulatory path.

The agency recommended that the gene therapy company conduct a prospective, randomized, double-blind, sham surgery-controlled study to proceed with its application.

In addition, the FDA seeks to align on the confirmatory study design prior to the BLA submission, including consideration of concurrent control on standard-of-care therapy instead of a sham procedure.

Phase 1/2 Data Supports Filing Strategy

In September 2025, uniQure released topline data from the pivotal Phase 1/2 study of AMT-130 for Huntington’s disease.

The study met its prespecified primary endpoint, with high-dose AMT-130 demonstrating a statistically significant slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS) at 36 months compared to a propensity score-matched external control.

Analyst Sees Signs Of More Flexible FDA

William Blair, in an analyst note on Wednesday, wrote, “Given Vinay Prasad's and Marty Makary's public opinion on this filing and CRL issuance, and now the positive uniQure regulatory update for AMT-130, we believe the current FDA, largely in caretaker mode, appears to be more flexible on regulatory paths for applications where concerns were previously raised.”

Prasad was reported to be involved in denying an expedited review path for AMT-130 gene therapy in Huntington’s disease, despite substantial patient and clinician lobbying.

QURE Stock Price Activity: uniQure shares were up 83.36% at $49.49 during premarket trading on Wednesday, according to Benzinga Pro data.

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