Did Sarepta complete the confirmatory trial for Exondys 51?
Not on the original timeline — the FDA set a November 2020 completion deadline that the company did not meet, illustrating a broader pattern in which accelerated approvals for desperate patient populations are politically difficult to withdraw.
Not on the original timeline. When the FDA granted accelerated approval, it required Sarepta to run a randomised controlled trial to verify that the drug actually slows disease progression. According to a review in the Annals of Internal Medicine, the FDA initially set a November 2020 deadline for completing that trial, which the company did not meet.
The same review noted that eteplirsen's approval was followed by approvals of other drugs in the same class via the same pathway, and that the category has been controversial because of questionable evidence, delays in post-approval testing and high US prices. Critics argue that once a drug for a desperate patient population is on the market, it becomes politically very difficult for the FDA to withdraw it, which weakens the incentive to complete confirmatory studies promptly.