Opdivo was granted an accelerated approval for patients with hepatocellular carcinoma, but the indication was pulled after follow-up data failed to show that the agent improved survival over another drug.
Opdivo (nivolumab) received an accelerated approval in 2017 to treat patients with hepatocellular carcinoma (HCC; the most common type of liver cancer) who have previously been treated with Nexavar (sorafenib). However, after more follow-up, the immunotherapy agent did not achieve statistical significance for overall survival, so the Food and Drug Administration (FDA) and Bristol Myers Squibb decided to withdraw the indication.
The original approval was based on findings from the phase 1/2 CheckMate-040 trial, which showed that Opdivo led to an 18.2% overall response rate (ORR), including 3.2% of patients who achieved a complete response, meaning that there was no detectable evidence of cancer after they received therapy.
But when the FDA reviewed further data in the confirmatory phase 3 CheckMate-459 clinical trial, Opdivo did not have a statistically significant increase in overall survival compared to Nexavar.
“We are disappointed by the position the Advisory Committee and the FDA have taken regarding the continued approval of Opdivo monotherapy as a treatment for HCC post-sorafenib. HCC is a complex and challenging disease, and for patients who are initially treated with sorafenib and either cannot tolerate treatment or whose disease progresses, immunotherapy is an important treatment option. For the past three and a half years, Opdivo monotherapy has been an important option that physicians have relied on to address this need and is currently the most commonly used therapy in the post-sorafenib setting,” said Jonathan Cheng, senior vice president and head of oncology development at Bristol Myers Squibb, in a statement.
Patients with HCC who are currently receiving single-agent Opdivo should talk to their health care teams about next best steps and can reach out to Bristol Myers Squibb for access and reimbursement questions.
Opdivo plus Yervoy (ipilimumab) is still FDA approved for this patient population and continues to be studied both alone and in combination with Yervoy for other types of cancers.
“Opdivo helped usher in an entirely new way to treat patients with this disease. We continue to support the FDA’s accelerated approval program, which has been integral to enabling people with difficult to treat cancers to gain access to certain safe and effective new therapies sooner,” Cheng said.
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