Investigational Gene Therapy for Huntington Disease Receives RMAT Designation from FDA to Accelerate Development

AMT-130 (uniQure; Lexington, MA), an investigational gene therapy for the treatment of Huntington disease (HD), has been granted Regenerative Medication Advanced Therapy (RMAT) designation by the Food and Drug Administration (FDA). AMT-130 combines an adeno-associated virus (AAV) vector with a gene-encoding microRNA to reduce the production of the disease-associated human huntingtin protein. The gene therapy is administered once via a MRI-guided, convection-enhanced, stereotactic neurosurgical procedure to the striatum. RMAT designation grants all the benefits of Breakthrough Therapy and Fast Track designation and is provided to therapies that address an unmet medical need for a serious condition by modifying, reversing, treating, or curing it. Such designation can allow for accelerated market approval based on surrogate or intermediate endpoints.

The FDA granted RMAT designation to AMT-130 based on an analysis of interim phase 1/2 clinical data, demonstrating that the gene therapy addresses a major unmet medical need for people with HD. The phase 1/2 clinical trial (NCT04120493) is evaluating the safety, tolerability, and efficacy of AMT-130 for the treatment of 26 participants with HD. The participant population was divided into a low-dose cohort (n=10) and a high-dose cohort (n=16), and participants were randomized to receive AMT-130 treatment (n=16) or a sham surgery (n=10). Enrollment for an additional cohort of 13 participants receiving dosing with AMT-130 in conjunction with perioperative immunosuppression is ongoing. According to a statement from uniQure, the company expects to provide updated interim data this year, including 3 years of follow-up for 29 AMT-130–treated participants.

“The RMAT designation, which was based on the comparison of the two-year AMT-130 data to a natural history cohort, marks a promising start to our FDA interactions,” stated Walid Abi-Saab, Chief Medical Officer of uniQure. “Importantly, RMAT designation allows for increased collaboration with the FDA to accelerate development, potentially facilitating earlier access for patients with life-threatening medical conditions.”