Revisiting Xenazine’s Extraordinary Review: A Study In FDA Flexibility
Unmet need is high in Huntington’s disease and the protracted review of Xenazine – the only drug cleared for the disease by FDA – suggests regulatory precedent for approval based on just one efficacy endpoint and with lots of development and safety baggage.
You may also be interested in...
Biogen and Eisai's surprise decision to advance Alzheimer’s antibody despite mixed clinical results could follow a similar path as pioneering neurodegenerative therapies Xenazine and Rilutek, or more recent Parkinson’s drug Nourianz – or could end with suspended development, like Kyndrisa.
The Danish drugmaker will pay $530 million upfront, but analysts believe it will not have to pay out the $129 million in contingent value rights payments.
The PBT2 compound boosts executive function in first Phase II trial, but fails all other efficacy endpoints; however, past regulatory experience suggests that with few treatment options, one endpoint can do the trick in hard-to-treat Huntington’s disease.