FDA orders phase 3 for Huntington's gene therapy, delaying uniQure's path to approval
TL;DR
The FDA rejected uniQure's plan to file AMT-130 using phase 1/2 data with an external control and demanded a prospective, randomized, sham-controlled phase 3 trial, dampening near-term approval hopes and triggering a strategic review by the company; uniQure will discuss trial designs with the FDA and add a four-year durability analysis to the phase 1/2 data.
- UniQure shares crash 40% as FDA rejects early approval path for Huntington's gene therapy Fierce Biotech
- The road ahead for uniQure: FDA says more data needed for AMT-130 gene therapy HDBuzz
- Opinion | The FDA shifts the goalposts on rare disease drugs The Washington Post
- UniQure Stock Drops 36% After Earnings. Bad News From the FDA Gets Even Worse. Barron's
- UniQure plans to seek approval for Huntington’s therapy still blocked by FDA statnews.com
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