HDSA, Help4HD, HD Reach, HDF and HDYO have joined forces on a petition directly to the FDA.
Key Asks of the FDA:
- Honor Your Previous Guidance: Fully uphold the previously agreed-upon accelerated approval pathway for AMT-130, including the use of external control data from natural history studies.
- Recognize the Urgency: Acknowledge the dire, unmet medical need in the HD community and act with the expediency that Breakthrough Therapy and RMAT designations demand.
- Expedite Review: Make the review and approval of AMT-130, and any other future HD therapies, a top priority to get this potentially life-altering therapy to patients without further delay.
Sign the Petition Today! Don’t forget to confirm your email to make it count.
