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Xpovio's US FDA Review: A Primer On How (Not) To Submit Real-World Data

 
• By Michael Cipriano

Karyopharm included a real-world data study to support its Xpovio NDA, but the US FDA deemed the study inadequate for an overall survival comparison to the pivotal STORM trial.

Drug Review Profile regular column

Real-world data (RWD) is still in its infancy at the US Food and Drug Administration, and sponsors are trying to navigate the landscape of how such data can support their drug applications. An inside look at an RWD study by Karyopharm Therapeutics Inc. to support the accelerated approval for its multiple myeloma drug Xpovio provides a cautionary tale on what not to do.

Xpovio got the agency nod 3 July 2019 in combination with dexamethasone for the treatment of adults with relapsed or refractory multiple myeloma who have received at least four prior...

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