uniQure Shares Rally as CBER Head Departs, AMT-130 Review Path May Open
FDA CBER head Dr. Vinay Prasad will exit in April after denying an expedited review pathway for uniQure’s AMT-130 Huntington’s gene therapy. uniQure shares rallied as investors anticipate smoother regulatory approval using single-arm studies and external controls.
1. Leadership Change at CBER
The U.S. FDA’s CBER director, Dr. Vinay Prasad, will leave his position in April after overseeing biologics reviews since May 2025. His tenure was marked by controversial decisions on gene therapies, vaccines and orphan drugs.
2. Impact on AMT-130 Review Pathway
Dr. Prasad previously denied an accelerated review for uniQure’s AMT-130 gene therapy for Huntington’s disease despite strong patient and clinician support. His departure is expected to ease use of single-arm studies and external control groups for regulatory filings.
3. Investor Reaction and Share Movement
uniQure shares have rallied following the announcement as investors anticipate a smoother approval process for AMT-130 and similar therapies. Market sentiment suggests reduced regulatory risk could enhance valuation of uniQure’s gene therapy pipeline.