The approval was based on data from the KEYNOTE-224 study, a single-arm trial in patients with HCC who had disease progression on or after sorafenib or were intolerant to sorafenib; had measurable disease; and had Child-Pugh class A liver impairment. Patients (N=104) were treated with Keytruda 200mg intravenously every 3 weeks until unacceptable toxicity, investigator-assessed confirmed disease progression, or completion of 24 months of therapy; the primary efficacy measures were overall response rate (ORR) and duration of response according to RECIST v1.1, modified to follow a maximum of 10 target lesions and a maximum of 5 target lesions per organ, as assessed by blinded independent central review.
Results showed an ORR of 17% (95% CI, 11–26), with 1% of patients having a complete response and 16% of patients having a partial response. A duration of response of ≥6 months and ≥12 months was observed in 89% and 56% of patients who responded to treatment (N=18), respectively; the median duration of exposure was 4.2 months.
Continued approval of Keytruda for HCC may be contingent upon verification and description of clinical benefit in confirmatory trials as the indication was approved under the accelerated approval pathway based on tumor response rate and durability of response.
Keytruda, a programmed death receptor-1 (PD-1) blocking antibody, is also approved for the treatment of melanoma, non-small cell lung cancer, head and neck squamous cell cancer, classical Hodgkin lymphoma, primary mediastinal large B-cell lymphoma, urothelial carcinoma, microsatellite instability-high cancer, gastric cancer, and cervical cancer.
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