AMT-130 four-year data extend Huntington’s disease signal as FDA evaluates uniQure BLA
uniQure reports four-year ifezuntirgene data showing slower Huntington’s disease progression as its gene therapy BLA moves through FDA review.
Pharma & Biotech
Pharma & Biotech
AMT-130 slowed Huntington’s progression by 75%. uniQure has now filed for FDA approval
uniQure has submitted AMT-130 for accelerated U.S. approval after three-year Phase 1/2 data showed a 75% slowing of Huntington’s disease progression against a matched natural-history control.