FDA Agrees to Accelerated Approval Pathway for Promising Huntington’s Disease Gene Therapy

A Potential Lifeline for Huntington’s Patients

Huntington’s disease, a genetic disorder caused by mutations in the HTT gene, leads to the production of a mutant huntingtin protein that progressively damages nerve cells in the brain. The gene therapy is designed to prevent the production of this harmful protein by targeting the gene’s mRNA, marking it for destruction. Administered directly into two brain regions most affected by the disease, this one-time therapeutic approach aims to slow or halt disease progression.

Key Efficacy Measures
The FDA’s agreement hinges on two primary measures of efficacy:

• • Composite Unified Huntington’s Disease Rating Scale (cUHDRS): Assesses disease severity and progression.
  • Neurofilament Light Chain (NfL) Levels: A marker of nerve damage found in cerebrospinal fluid.

Interim analyses of the trials revealed promising results: an 80% reduction in disease progression, as measured by cUHDRS, and an 11% decrease in NfL levels compared with untreated patients.

Accelerated Approval Pathway: Speeding Access to Treatment

The accelerated approval pathway allows conditional FDA authorization based on early evidence of efficacy, significantly reducing the time to licensure. According to uniQure CEO Matt Kapusta, this approach could shorten the approval timeline by approximately five years.

“This is an important milestone for the Huntington’s disease community as it puts us on the most rapid and efficient pathway to deliver a potentially life-changing therapy,” said Dr. Walid Abi-Saab, uniQure’s Chief Medical Officer.

Regulatory Progress and Next Steps

The therapy is currently undergoing two Phase 1/2 trials in the U.S. (NCT04120493) and Europe (NCT05243017). The FDA confirmed that the data from these studies, supplemented by external control group comparisons, can serve as the basis for accelerated approval. An additional registrational trial will not be required.

In preparation for a regulatory submission, uniQure expects to finalize discussions with the FDA in early 2025. The company is also completing dosing for a third group of trial participants, aimed at assessing the impact of immunosuppressive therapy on the treatment’s safety.

Recognition and Designations
The therapy has received multiple regulatory designations to expedite its development:

• • Regenerative Medicine Advanced Therapy (RMAT): Allows frequent FDA engagement to facilitate development.
  • Orphan Drug Designation: In the U.S. and Europe for rare diseases.
  • Fast Track Designation: To speed up clinical development and review.

Looking Ahead

uniQure is set to release preliminary safety data for the immunosuppression group and three-year efficacy updates from earlier trial participants in 2025. These findings will further inform the FDA’s evaluation of the therapy’s potential as a transformative treatment for Huntington’s disease.

As the Huntington’s community awaits this breakthrough, the promise of gene therapy offers new hope for slowing or stopping the progression of this devastating neurodegenerative disorder.

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