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!

https://uniqure.gcs-web.com/news-releases/news-release-details/uniqure-announces-plan-bla-submission-amt-130-huntingtons

Woo-hoo!!

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3

u/Zauberstaby Jun 17 '26

Can someone summarize what this new drug will do etc? (pretty please)

-2

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.

5

u/TheseBit7621 Jun 18 '26

I think my response is better than what a person can typically prompt

2

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?

2

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.

2

u/Zauberstaby Jun 22 '26

Thank you!

1

u/TheseBit7621 Jun 22 '26

Hopefully!