FDA Grants Accelerated Approval to Keytruda for Hepatocellular Carcinoma Previously Treated with Sorafenib

Recently, Merck Sharp & Dohme (MSD) announced that the U.S. Food and Drug Administration (FDA) has approved a new indication for its PD-1 cancer immunotherapy Keytruda (Chinese brand name: Keytruda; generic name: pembrolizumab), for use in patients with hepatocellular carcinoma (HCC) who have previously been treated with the targeted drug sorafenib. This marks the 14th indication for Keytruda under FDA regulation in the United States. The approval was granted based on accelerated approval criteria, relying on tumor response rate and durability of response data. Further approval will depend on the verification and description of clinical benefits in confirmatory clinical studies.

It is worth noting that at the end of last month, the FDA also approved Keytruda in combination with chemotherapy (carboplatin plus paclitaxel or nab-paclitaxel) for the first-line treatment of patients with metastatic squamous non-small cell lung cancer (squamous NSCLC), regardless of tumor PD-L1 expression status. This approval makes Keytruda the first PD-1 tumor immunotherapy for the first-line treatment of squamous NSCLC irrespective of tumor PD-L1 expression status.

The approval of Keytruda for the indication of hepatocellular carcinoma (HCC) was based on data from the KEYNOTE-224 study. This was a single-arm, open-label study that enrolled 104 patients with HCC who had disease progression during or after sorafenib treatment, or who were intolerant to sorafenib, to evaluate the efficacy and safety of Keytruda treatment.

Data showed that the overall response rate (ORR) with Keytruda treatment was 17% (95% CI: 11–26), with a complete response rate of 1% and a partial response rate of 16%. Among patients who achieved disease response (n=18), 89% had a duration of response (DOR) of 6 months or longer, and 56% had a DOR of 12 months or longer.

The study’s principal investigator, Director of Liver Cancer Research at Massachusetts General Hospital, HarvardMedicineAndrew X. Zhu, MD, Professor of Medicine at the hospital, stated that hepatocellular carcinoma (HCC) is the most common type of cancer in adults. Despite some progress in recent years, treatment options for advanced recurrent disease remain limited. The current approval of Keytruda is highly significant, as it provides a new therapeutic option for patients with HCC who have previously been treated with sorafenib.

Dr. Scot Ebbinghaus, Vice President of Clinical Research at MSD Research Laboratories, stated that the approval of Keytruda for advanced hepatocellular carcinoma (HCC) marks the second FDA approval this year for the company’s oncology portfolio in the treatment of HCC, underscoring its steadfast commitment to providing new therapeutic options for historically challenging cancers. The company looks forward to continuing to advance HCC research to help more patients affected by this disease.

Globally, liver cancer is the second leading cause of cancer-related deaths, with 780,000 new cases and approximately 750,000 deaths annually. Hepatocellular carcinoma (HCC) accounts for 90% of primary liver cancer cases. Early-stage HCC can be treated through various modalities, including surgical resection, radiofrequency ablation, percutaneous ethanol injection, and transarterial chemoembolization. However, treatment options for unresectable HCC are very limited, resulting in a poor prognosis and representing a significant unmet medical need. Clinically, Bayer’s targeted anticancer drug Nexavar (generic name: sorafenib) is the standard first-line molecular targeted therapy for unresectable HCC.

Liver cancer exhibits significant regional variation, with the majority of cases occurring in Asia, primarily in China, followed by Africa. Hepatocellular carcinoma (HCC) is associated with chronic liver disease, particularly cirrhosis. The main causes of cirrhosis include hepatitis B virus (HBV) and hepatitis C virus (HCV) infections. However, according to a recent survey, non-HBV/non-HCV-related hepatocellular carcinoma is on the rise.

Source: Bioon