FDA D.I.S.C.O. Burst Edition: FDA approval of Keytruda (pembrolizumab) as adjuvant treatment for non-small cell lung cancer
Regulatory approval: On January 26, 2023, the FDA approved pembrolizumab (Keytruda) as adjuvant treatment for adult patients with stage IB, II, or IIIA non-small cell lung cancer following complete resection and platinum-based chemotherapy. The indication was limited to patients who received prior adjuvant chemotherapy. Approval was based on KEYNOTE-091, which met its primary endpoint of investigator-assessed disease-free survival in the overall population; an exploratory subgroup without adjuvant chemotherapy showed a disease-free survival hazard ratio of 1.25.
FDA approval (Jan 26, 2023): adjuvant pembrolizumab in resected NSCLC
The US FDA approved pembrolizumab (Keytruda) on January 26, 2023 for adjuvant treatment in adult patients with stage IB, II, or IIIA non-small cell lung cancer (NSCLC) following resection and platinum-based chemotherapy. The FDA limited the indication to patients who received prior adjuvant chemotherapy.
Efficacy evidence: Approval was based on KEYNOTE-091, a multicenter, randomized, triple-blind, placebo-controlled trial. Patients had not received neoadjuvant radiotherapy or chemotherapy. Participants were randomized 1:1 to pembrolizumab 200 mg IV every 3 weeks or placebo for up to 1 year. Of 1177 randomized patients, 1010 (86%) received adjuvant platinum-based chemotherapy after complete resection. The primary endpoint was investigator-assessed disease-free survival (DFS); the trial met its primary endpoint with a statistically significant DFS improvement in the overall population. In an exploratory subgroup of 167 patients who did not receive adjuvant chemotherapy, the DFS hazard ratio was 1.25. Among those who received adjuvant chemotherapy, median DFS was 58.7 months vs 34.9 months (pembrolizumab vs placebo), HR 0.73.
Safety: Adverse reactions were generally similar to prior pembrolizumab experience in NSCLC, with exceptions including hypothyroidism, hyperthyroidism, and pneumonitis; two fatal myocarditis events occurred.
The review was conducted under Project Orbis; reviews at other agencies were ongoing.