Bristol Myers Squibb disclosed that in consultation with the FDA it decided to withdraw the small-cell lung cancer (SCLC) indication for Opdivo (nivolumab) in the US. The PD-1 immune checkpoint inhibitor had been granted accelerated approval  in 2018 for the treatment of patients with SCLC whose disease has progressed after platinum-based chemotherapy and at least one other line of therapy.
The authorisation was based on surrogate endpoints from the Phase I/II CheckMate -032 trial of patients with advanced or metastatic solid tumours, with Opdivo demonstrating encouraging response rates and duration of response in SCLC. Specifically, 12% of patients with SCLC responded to treatment with Opdivo, regardless of PD-L1 expression, with 12 patients having a partial response and one subject a complete response.
However, subsequent confirmatory studies in different treatment settings failed to meet their primary endpoints. In the Phase III CheckMate -331 trial  of patients with SCLC who relapsed following platinum-based chemotherapy, Opdivo failed to significantly prolong overall survival (OS) versus current standard of care. Meanwhile, in the CheckMate -451 study of patients with extensive-stage SCLC who did not experience disease progression after first-line platinum-based chemotherapy, Bristol Myers Squibb's therapy in combination with the CTLA-4 inhibitor Yervoy (ipilimumab) failed to significantly extend OS versus placebo.
Bristol Myers Squibb explained that the decision to pull the SCLC indication was taken "in accordance with the [FDA's] standard procedures for evaluating accelerated approvals that have not met their post-marketing requirements and as part of a broader industry-wide evaluation." Opdivo generated sales  of $1.8 billion in the third quarter of 2020, down 2% year-on-year.