FDA undecided on UniQure Huntington’s therapy; no proven benefit blocks marketing filing
TL;DR
An FDA official indicated UniQure’s Huntington’s disease therapy showed no demonstrated benefit based on current data, preventing a marketing application; the company disputes the assessment but hopes to align trial parameters for a potential future study.
Topics:businesshealthcare#drug-development#fda#healthcare#huntingtons-disease#regulatory-decision#uniqure
- FDA is ‘not convinced’ UniQure’s Huntington’s therapy has benefit, senior official says statnews.com
- UniQure Eyes Phase 3 for Huntington’s Gene Therapy Despite Makary’s Apparent Criticism BioSpace
- Opinion | The FDA shifts the goalposts on rare disease drugs The Washington Post
- The road ahead for uniQure: FDA says more data needed for AMT-130 gene therapy HDBuzz
- FDA holds line on uniQure filing for Huntington's gene therapy FirstWord Pharma
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