Accelerated Approvals Under the Microscope
The accelerated approval program has come under scrutiny recently, with notable market withdrawals and failed confirmatory trials raising questions regarding the risk-benefit proposition.
Most recently, Pfizer withdrew sickle cell disease therapy Oxbryta from worldwide markets five years after its accelerated approval, after new data showed a higher risk of deaths and complications in treated patients. And earlier this year, Biogen pulled Aduhelm from the shelves after the Alzheimer’s drug failed to gain traction. Aduhelm’s accelerated approval, based on its ability to reduce amyloid in the brains of Alzheimer’s patients, was among the most controversial in recent history.
Meanwhile, Sarepta’s Elevidys, which won accelerated approval in June 2023 as the first gene therapy for Duchenne muscular dystrophy, failed its confirmatory trial four months later, adding fuel to already simmering questions over the treatment’s efficacy. Despite this setback, Elevidys won full FDA approval in June 2024.
In order to improve the program, experts recently told BioSpace that tighter completion timelines for confirmatory trials and a better understanding of which biomarkers should be used as surrogate endpoints are key.
