The US Food and Drug Administration’s regulatory nod for Epizyme Inc.’s epithelioid sarcoma drug Tazverik (tazemetostat) reflects the careful balancing act agency staff perform each time a drug comes through the accelerated approval pathway – deciding whether it should be made available for a desperate patient population in the face of limited trial data, and at the risk the sponsor will never be able to definitively confirm clinical benefit.
In the case of tazemetostat, reviewers’ doubts about the magnitude of the efficacy benefit were overridden by the agency’s emphasis on flexibility under the expedited
The new drug application’s clinical reviewer and cross-disciplinary team leader recommended against accelerated approval of the methyltransferase inhibitor, finding the modest overall response rates in the uncontrolled clinical trial cohorts did not provide substantial evidence of a clinically meaningful improvement over
