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Revisiting Xenazine’s Extraordinary Review: A Study In FDA Flexibility

 
• By Emily Hayes

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.

FDA’s 2008 approval of Valeant Pharmaceuticals International Inc./Lundbeck Inc. ’s Xenazine (tetrabenazine) – still the only drug currently cleared in the U.S. for Huntington’s disease – illustrates the agency’s flexibility on efficacy endpoints in the space.

There could be some lessons learned for Prana Biotechnology Ltd. ’s PBT2, which succeeded in demonstrating a statistically significant improvement on only one efficacy measure of executive function in...

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