Tazverik Accelerated Approval: Disease Rarity And Unmet Need Won Out Over Efficacy Concerns
US FDA reviewers recommended against accelerated approval of Epizyme’s tazemetostat for epithelioid sarcoma, finding the modest overall response rates were not substantial evidence of an improvement over available therapies; they were overruled by senior oncology review staff, who cited a unanimous ODAC endorsement and the regulatory flexibility encouraged for rare diseases.
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Sponsor had hoped to use retrospective case review study as an external control arm to support regular approval of tazemetostat; US FDA not only rejected this idea, it found the study design so flawed that it did not consider the results in its efficacy assessment for accelerated approval.
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.)