FDA U-turn stalls promising Huntington's gene therapy from uniQure

The FDA has reversed course on uniQure’s AMT-130 gene therapy, no longer supporting a fast-track approval based on existing phase 1/2 data.

Why it matters: AMT-130 showed a 75% slowdown in Huntington’s progression over 3 years, offering rare hope for a condition with no disease-modifying treatments. The FDA's decision delays potential access for patients and signals a broader shift in regulatory scrutiny of gene therapies.

Backstory: AMT-130, a one-time gene therapy, was touted as a breakthrough after showing major clinical benefits in early trials. Based on these results, the FDA had initially agreed to consider accelerated approval using an external control group comparison. The reversal came during a recent pre-biologics license application meeting, catching uniQure off guard.

Big picture: The decision reflects the FDA’s tougher stance under Center for Biologics Evaluation and Research Director Vinay Prasad. Other biotechs, like Capricor and Replimune, have also faced unexpected rejections despite following prior FDA guidance.

Zoom in: uniQure’s stock surged 300% with the release of initial data but plummeted 66% after the FDA shift. Patient advocates may challenge the FDA, echoing past pushback that influenced decisions on other gene therapies like Sarepta's Elevidys.