UniQure Files for Regulatory Approval of Huntington’s Gene Therapy

Rare Daily Staff

UniQure said it has submitted a biologics license application to the U.S. Food and Drug Administration seeking accelerated approval of its experimental gene therapy, AMT-130, for Huntington’s disease, a rare neurodegenerative condition.

The company also submitted a marketing authorization application to the U.K. Medicines and Healthcare products Regulatory Agency.

The applications are supported by three-year data from UniQure’s phase 1/2 program, which compared treated patients with a propensity-score-matched external control group drawn from the Enroll-HD natural-history database. The company said the analysis showed that AMT-130 slowed disease progression, although the release did not provide detailed efficacy or safety results.

Huntington’s disease is an inherited neurodegenerative condition caused by a disease-causing expansion in the huntingtin gene. It progressively affects movement, cognition and behavior. No approved treatment has been shown to delay disease onset or slow its progression.

AMT-130 is designed to silence the huntingtin gene, including a potentially toxic exon 1 protein fragment. It is administered once through MRI-guided neurosurgical infusion directly into the striatum, a brain region affected early in Huntington’s disease.

The FDA has granted the therapy Breakthrough Therapy, Regenerative Medicine Advanced Therapy and Fast Track designations. It is the first Huntington’s disease treatment candidate to receive both Breakthrough Therapy and RMAT designations.

The company’s clinical program includes a randomized U.S. phase 1/2 study and an open-label European study. In the U.S. trial, $26$ patients received either low-dose or high-dose AMT-130 or underwent a sham procedure; four participants in the control group later crossed over to treatment. The European study enrolled $13$ patients.

Additional cohorts have evaluated AMT-130 with immunosuppression and in patients with lower striatal volumes. UniQure said it plans to report a four-year analysis from the ongoing phase 1/2 studies before the end of the third quarter.