FDA Accepts Bristol Myers Squibb's sBLA for First-Line Treatment of Unresectable Hepatocellular Carcinoma

Bristol Myers Squibb has announced that the U.S. Food and Drug Administration (FDA) has accepted a supplemental Biologics License Application (sBLA) for their drugs Opdivo® (nivolumab) and Yervoy® (ipilimumab). This combination is proposed as a first-line treatment for adult patients with unresectable hepatocellular carcinoma (HCC). The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of April 21, 2025, for this application.

Hepatocellular carcinoma, the most prevalent form of liver cancer, is frequently diagnosed at an advanced stage when surgical options are not viable. Over the past decade, the incidence of HCC in the United States has risen, underscoring the urgent need for new treatment alternatives.

The sBLA submission is buoyed by findings from the Phase 3 CheckMate -9DW trial, which indicated that the combination of Opdivo and Yervoy significantly improved overall survival rates compared to the use of lenvatinib or sorafenib, two existing treatment options. Currently, Opdivo plus Yervoy is approved as a second-line treatment for advanced HCC. The new data suggest that this combination could serve as an effective first-line therapy as well. The safety profile of the Opdivo and Yervoy combination aligns with previous data and presents no new safety concerns. These results were also shared at the 2024 American Society of Oncology (ASCO) Annual Meeting.

The CheckMate -9DW study was a Phase 3, randomized, open-label trial that compared the efficacy of Opdivo and Yervoy against lenvatinib or sorafenib monotherapy in patients with unresectable or advanced HCC who had not undergone prior systemic therapy. A total of 668 patients participated in the trial, receiving either the combination treatment or one of the monotherapies. The trial’s primary endpoint was overall survival, with secondary objectives including the objective response rate and time to symptom deterioration.

Liver cancer is the third leading cause of cancer-related deaths globally, with HCC accounting for 75%-85% of all liver cancer cases. Typically diagnosed at an advanced stage, HCC has limited effective treatment options, often resulting in poor outcomes. Recurrence within five years is common, affecting up to 70% of patients, particularly those at high risk post-surgery or ablation. While hepatitis B and C viruses are primary causes of HCC, increasing instances of metabolic syndrome and nonalcoholic steatohepatitis (NASH) are contributing to rising HCC rates.

Bristol Myers Squibb is dedicated to transforming patients' lives through innovative scientific research. The company aims to develop medicines that not only improve but potentially cure various forms of cancer. With a comprehensive understanding of human biology and advanced technological capabilities, Bristol Myers Squibb is exploring new dimensions in personalized medicine and using data-driven insights to refine their focus on cancer treatment.

Opdivo, a programmed death-1 (PD-1) immune checkpoint inhibitor, leverages the body's immune system to combat cancer. This drug is approved for multiple cancer types worldwide and has treated over 35,000 patients through various clinical trials. Opdivo’s global development program, built on Bristol Myers Squibb’s expertise in Immuno-Oncology, includes numerous clinical trials across different phases and tumor types. Since its first regulatory approval in July 2014, Opdivo has been approved in over 65 countries.

Yervoy is a monoclonal antibody targeting the cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4) to enhance T-cell activation and proliferation, aiding in the body’s anti-tumor response. Approved initially in 2011 for metastatic melanoma, Yervoy is now authorized for use in more than 50 countries for various tumor types.

Bristol Myers Squibb continues to explore new treatments and combinations to improve cancer care, aiming to provide better outcomes for patients globally.