uniQure jumps 70% after FDA backs three-year data for AMT-130 BLA
QURE•uniQure stock surged 70% pre-market after the FDA agreed to base an accelerated BLA on three-year Phase I/II data for AMT-130 Huntington’s therapy. The company plans a Q3 2026 submission and holds RMAT, Breakthrough Therapy and Fast Track designations.
1. FDA Endorses Three-Year Data Basis
On June 17, the FDA communicated that data from uniQure’s ongoing Phase I/II AMT-130 trial, following three years of follow-up, will serve as the primary dataset for a Biologics License Application, shifting from its March recommendation for a larger randomized study.
2. Filing Schedule and Regulatory Designations
uniQure plans to submit the BLA for AMT-130 in the third quarter of 2026; the therapy has already received RMAT, Breakthrough Therapy and Fast Track designations to expedite development and review processes.
3. U.K. MHRA Filing Plans
The company intends to seek approval from the U.K. Medicines and Healthcare products Regulatory Agency in Q3 2026, supported by three-year trial data demonstrating a 75% reduction in Huntington’s disease progression.
4. Market Reaction
Shares of the company jumped over 70% in pre-market trading and reached their highest levels in more than seven months, reflecting renewed investor optimism around the gene therapy’s regulatory prospects.
