Tazverik Withdrawal Shows How FDA Accelerated Approval Works and Ends

FDA has withdrawn approval of Tazverik (tazemetostat), the cancer drug held by Epizyme, an Ipsen company, in a notice scheduled for publication in the Federal Register on June 22, 2026. All three of the drug's indications had been granted under FDA's accelerated approval pathway, and the notice says the trial meant to confirm the benefit in follicular lymphoma instead showed an increased rate of second primary malignancies.

The case runs through each stage of accelerated approval: the early approval, the confirmatory trial and the withdrawal.

Approval on a predicted benefit

FDA's Accelerated Approval Program page, as archived in early June 2026, says the pathway allows earlier approval of drugs that treat serious conditions and fill an unmet medical need, based on a surrogate endpoint. It defines a surrogate endpoint as a marker, such as a laboratory measurement or radiographic image, "that is thought to predict clinical benefit but is not itself a measure of clinical benefit."

Companies must still run studies to confirm the expected benefit. If a confirmatory trial shows the drug provides a clinical benefit, FDA grants traditional approval. If it does not, the page says, FDA "has regulatory procedures in place that could lead to removing the drug from the market."

Tazverik followed that route. According to the notice, FDA approved it on Jan. 23, 2020, for certain patients aged 16 and older with metastatic or locally advanced epithelioid sarcoma, and on June 18, 2020, for two groups of adults with relapsed or refractory follicular lymphoma. All three approvals required Epizyme to conduct postmarketing trials "to verify and describe the clinical benefit."

When the confirmatory trial goes wrong

The confirmatory trial for the lymphoma indications was SYMPHONY-1, which compared Tazverik plus lenalidomide and rituximab with those two drugs plus placebo. The notice says Epizyme told FDA on March 6, 2026, of an increased rate of second primary malignancies in the Tazverik arm and, at the same time, that it was withdrawing the drug from the U.S. market.

FDA met the company on April 16. On April 30, Epizyme asked FDA to withdraw the application and waived the expedited withdrawal procedures, which FDA had asked it to do. The notice cites "the increased rate of second primary malignancies in the confirmatory trial" among the reasons for the withdrawal, and says distributing the drug without an approved application is illegal.

The expedited withdrawal procedures

Congress rewrote the withdrawal rules in the Consolidated Appropriations Act, 2023. FDA's December 2024 draft guidance on accelerated approval says the agency may use expedited procedures to withdraw an accelerated approval if:

• the sponsor fails to conduct a required postapproval study with due diligence;
• a required confirmatory study fails to verify clinical benefit;
• other evidence shows the product is not safe or effective under its conditions of use;
• the sponsor disseminates false or misleading promotional materials.

Those procedures include notice and an explanation to the sponsor, a meeting with the Commissioner or a designee, a written appeal, a public comment period and, if the sponsor asks and none has already advised on the matter, an advisory committee meeting. The guidance says discussions with FDA may instead lead a sponsor to request withdrawal voluntarily, the route Epizyme took.

The same law gave FDA other tools. The guidance says the agency may require confirmatory studies to be underway before accelerated approval is granted, and sponsors must report on their progress about every 180 days.