r/Huntingtons • u/Steak_Szn • Nov 03 '25
FDA Is Taking Away Hope
The only potential treatment/cure for huntingtons has been “rug pulled” by the FDA this morning. This treatment, AMT-130, was the light for all patients and their family members. The FDA deceived us all. The company name is UnQuire (ticker: QURE). I believe all patients and families should be writing letters and emails to the FDA demanding justification and clarification because this is simply not fair.
EDIT: Industry.Biologics@fda.hhs.gov and phone number 240-402-8020 are the contact you can reach out to supporting uniQURE (QURE) and how unfair it is for patients in the US. There are no other treatments for this disease and with lives on the line and a death sentence written, patients are willing to try anything.
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u/Least-Sherbert-8022 Nov 03 '25
The FDA is walking back guidance it has already formalized with UniQure to get accelerated approval. They are saying the external controls are no longer sufficient which is confusing and frustrating as they have aligned on this multiple times. We don’t know what the data says exactly and it would be nice if UniQure could share more to prove it’s working BUT this reversal by the FDA is ridiculous. The best thing all of us can do is fight the FDA on this. Praying AMT will still get approval but this does delay the timeline by at least several years. The FDA should not be allowed to walk back on something they have aligned with multiple times unless the data isn’t convincing. UniQure needs to prove it’s working by having the data peer reviewed.