FDA Committee Votes to Withdraw Two Accelerated Approvals of Cancer Treatments

The U.S. Food and Drug Administration’s (FDA’s) Oncologic Drugs Advisory Committee (ODAC) recommended withdrawing two of six accelerated approvals of cancer treatments that it reevaluated in a recent meeting, citing the failure to verify benefit in follow-up trials.

The committee voted 6-2 to withdraw the accelerated approval of pembrolizumab for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction adenocarcinoma whose tumors express PD-L1, with disease progression on or after two or more prior lines of therapy. In follow-up trials, pembrolizumab had a low overall response rate of 13% in patients with third-line gastric cancer.

“Would we grant this indication at this time?” Richard Pazdur, MD, head of FDA’s Oncology Center of Excellence, asked during the meeting. “The definitive answer is no.”

In addition, committee members voted 5-4 to withdraw the accelerated approval of nivolumab as a single agent for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. Nivolumab’s approval in combination with ipilimumab to treat HCC will stand, as the combination has shown higher overall response rates compared with nivolumab monotherapy (32% vs. 14%) in clinical trials.

Prior to the ODAC meeting, only 5% of accelerated approvals for cancer drugs have been withdrawn since the program’s inception in 1992.

Sources: RAPS.org, April 30, 2021; Yau T, Kang Y, Kim T, et al. Efficacy and safety of nivolumab plus ipilimumab in patients with advanced hepatocellular carcinoma previously treated with sorafenib: the CheckMate 040 randomized clinical trialJAMA Oncol. 2020;6(11):e204564.