Given the rarity of epithelioid sarcoma, ‘the possibility exists that this trial will not be completed,’ Division of Oncology 3 acting director Steven Lemery said in recommending approval; confirmatory trial is not expected to complete until 2029, and FDA reviewers suggest it may be underpowered to show an effect on progression-free survival. (Part 2 of our Drug Review Profile on the product.)
Concerns about the confirmatory trial's feasibility and utility hung over the tazemetostat approval decision. • Source: Shutterstock
The US Food and Drug Administration granted Epizyme Inc.'s Tazverik (tazemetostat) accelerated approval for epithelioid sarcoma despite concerns that the required confirmatory trial may not provide useful information or even be completed.
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The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.
Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.
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