Stand Up Speak Up 4 HD: Urging Accelerated FDA Access to Life-Changing HD Therapies and Treatments

FDA Contacts:

Emails

OCOD@FDA.HHS.gov

patientaffairs@fda.hhs.gov

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Food and Drug Administration

Center for Biologics Evaluation and Research (CBER)

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Silver Spring, MD 20993-0002

Template One:

Subject: Urgent: FDA Reconsider Accelerated Approval for uniQure’s AMT-130 Therapy in Huntington’s Disease

Dear FDA CBER -

We face a critical moment in the fight against Huntington’s disease. The FDA’s recent hesitation in allowing uniQure to submit a BLA for its groundbreaking gene therapy, AMT-130, poses an unacceptable delay in access to a treatment that could significantly slow disease progression.

The FDA has previously agreed that:

·       Data from uniQure’s Phase 1/2 trials using a natural history external control group is a scientifically valid basis for potential accelerated approval.

·       The Composite Unified Huntington’s Disease Rating Scale (cUHDRS) can serve as an intermediate clinical endpoint.

·       Biomarkers like neurofilament light chain (NfL) reductions provide supportive evidence of efficacy.

Accelerated approval is designed to provide earlier access to promising therapies for serious conditions like HD, where waiting for traditional, lengthy trials is not an option. The external placebo (natural history) control group is a well-recognized and ethical approach in rare, devastating diseases where randomized placebo controls are challenging.

Without the consideration for potential accelerated approval by the FDA, people with HD will continue to suffer and die earlier than if a therapy or treatment were available. And those at risk of inheriting HD will live and suffer without hope.

We ask that the FDA:

·       Fully honor the previously agreed accelerated approval pathway using external control data.

·       Recognize the urgent unmet medical need for HD treatments.

·       Expedite the review and approval process to make AMT-130 available to patients without further delay.

Huntington’s patients and families are in urgent need of timely access to potentially life-changing therapies. Every minute counts.

We respectfully urge the FDA to reconsider and uphold accelerated approval of uniQure’s AMT-130.

Respectfully,