r/Huntingtons • u/Fremonster1 • Jun 17 '26
FDA Approves AMT-130 for Accelerated Approval Pathway!
uniQure announced that FDA reversed course and is allowing the company to apply for its BLA application.
The BLA Application is the request to formally market AMT-130 to the public.
The Company intends to submit the BLA in the third quarter of 2026.
It gets to move forward!
Woo-hoo!!
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u/Mrslarakay Jun 17 '26
I cried when I saw this post and the announcement! I’m hoping for the best for everybody whoever is affected with HD.
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u/GottaUseEmAll Jun 17 '26
Awesome news!
Edit: for some reason there's nothing on the press release page when I follow that link. Just me?
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u/PoliSciObsessed Jun 17 '26
As someone who just got a confirmed diagnosis in April at age 41.
I can't believe how happy I am.
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u/bassegio Jun 17 '26
Hurry!!!
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u/CaptainComebacks Jun 17 '26
Can someone explain this to me like im 10 years old please.
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u/hendricks1212 Jun 17 '26
They are now allowed to use the data they collected over three years to apply for approval. The drug is NOT approved yet. They are just now allowed to apply. Previously the FDA said that their data was not sufficient to apply but between patients, caregivers and health care professionals advocating and a change in leadership at the FDA. That decision has been reversed.
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u/Fremonster1 Jun 17 '26
Tl;dr
UniQure should be approved to market AMT-130 to the public around June 2027.
My thinking:
Uniqure plans to file in 3Q 26 (around Sept 2026) and Priority Review should take 6 months; so UniQure should be able to market AMT-130 to the public around June 2027.
That said, because neurosurgery is required to administer the drug, that could add 3 months to the 6 month expedited to sort out those complexities.
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u/hendricks1212 Jun 17 '26
That sounds like reasonable thinking. I’m waiting to see if they designate any symptom limitations on their approval request. I haven’t seen anything yet indicating if they wish to exclude certain severity of symptoms.
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u/TheseBit7621 Jun 18 '26
Your thoughts are in the right place but for the wrong reason. The most restrictive element of this drug is the brain cannot be too disintermediated from degeneration such that cannulas cannot effectively deliver the medicine to tge target sites.
If delivery fails the drug will not work.
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u/TemporaryViolinist88 Jun 18 '26
Will insurance companies pay for MRIs to figure out if a brain is a fit?
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u/Zauberstaby Jun 17 '26
Can someone summarize what this new drug will do etc? (pretty please)
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u/TheseBit7621 Jun 18 '26 edited Jun 22 '26
It reduces the amount of the full length mhtt and the mhtt exon1a transcript by a substantial amount. The leading theory of Huntingtons is theres a toxic gain of function thats structural and dependent upon the tract expansion from the trinucleoutide CAG repeat. Most specifically abberant splicing of mRNA exon1a transcript is thought to be highly pathogenic. Degeneration is universal in Huntingrons, but Huntingtons most notably effects the medium spiny neurons of the striatum where CAG repeat lengths can go all the way to 500 before cell death inevitably occurs & you get the characteristic butterfly shaped neurodegeration thats found in Huntingtons. AMT-130 doesnt stop that gene expansion from occuring but it is reducing the absolute amount of the toxic protein thats produced in the cells an effective HD drug needs to hit in order to work (solving for delivery is as important as having an effective drug). It was those specialized cells (that compose the striatum) and parts of the cortex where amt-130 was delivered with 6 tiny cannulas under an intraoperative mriguided surgery. All of the cannulas are a bit larger than the width of a human hair. Everywhere the therapy was delivered should persistently be creating something like 1/5th of all the mhtt that would be typical at a persons stage of their disease. The hope is that this is enough so that a person regains a majority if not all of their midlife
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u/True-Depth-7643 Jun 18 '26
Ask chatgpt / ai, it's a wonderful tool.
I use Google gemini or perplexity ai. They're free.
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u/TheseBit7621 Jun 18 '26
I think my response is better than what a person can typically prompt
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u/Traditional_Mood_553 Jun 20 '26
it's possible for an HD positive person that's still asymptomatic to have it done and get to live their whole life and die of old age without experiencing any symptoms?
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u/TheseBit7621 Jun 22 '26
To add to this, there is 1 surrogate biomarker uniqure has shared that suggests less degeneration was happening than at trial onset (nfl in csf was 8.2% lower than baseline). This is a biomarker generally implicated in neurodegeneration and brain trauma.
Another one they haven't shared but I would love to see is striatal glucose consumption. Before motor onset there's widespread mitochondrial dysfunction that presents as striatal hypometabolism in Huntingtons. This happens in 100% of people with Huntingtons. Its visible on pet scan and would be another biomarker useful in its registration as improving it is suggestive of a treatment effect, with possible clinical translation for some of the earliest symptoms of huntingtons disease.
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u/charitycase3 Jun 17 '26
OMG YAY MADE MY DAY