The Food and Drug Administration will accept uniQure’s three-year AMT-130 data as the primary basis for an accelerated approval filing, but external controls, irreversible brain delivery and confirmatory evidence remain decisive risks.
Explore whether uniQure’s AMT-260 gene therapy can rival epilepsy surgery after early seizure reductions in a small first-in-human cohort.
Find out how uniQure’s AMT-260 early epilepsy data could shape the next test for brain-delivered gene therapy.
Explore why the FDA reopened uniQure’s AMT-130 approval path and what evidence, surgical and confirmatory trial risks remain before review.