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Lynne Mitchell
As therapies for rare and neurological diseases earn accelerated approval, experts laud the program’s intent while remaining concerned about confirmatory trials and clinical efficacy, especially as products greenlit under this pathway are pulled from the market.
Last month, Pfizer’s sickle cell disease therapy
Oxbryta
, which won accelerated approval in 2019, was
pulled from market
due to risk of death and other complications, illustrating the risk of greenlighting ther