13 November 2019 - - The US Food and Drug Administration has accepted its supplemental Biologics License Application and granted Breakthrough Therapy Designation for Opdivo (nivolumab) in combination with Yervoy (ipilimumab) for the treatment of patients with advanced hepatocellular carcinoma previously treated with sorafenib, US-based biopharmaceutical company Bristol-Myers Squibb Company (NYSE: BMY) said.
The FDA granted the application Priority Review with a Prescription Drug User Fee Act (PDUFA) goal date of March 10, 2020.
This application is based on data from the Opdivo plus Yervoy cohort of the Phase 1/2 CheckMate -040 study evaluating the Immuno-Oncology combination in patients with advanced HCC previously treated with sorafenib.
Data from this study were presented at the 2019 American Society of Clinical Oncology annual meeting.
The FDA's Breakthrough Therapy Designation is a process intended to enable timely patient access by expediting the development and review of medicines for serious conditions where preliminary clinical evidence indicates a substantial improvement over available therapies on one or more clinically significant endpoints.
CheckMate -040 (NCT01658878) is an ongoing Phase 1/2, open-label, multi-cohort study investigating Opdivo or Opdivo-based combinations in patients with advanced HCC with and without chronic viral hepatitis who are naïve, intolerant to or who have progressed during sorafenib therapy.
The Opdivo plus Yervoy cohort of CheckMate -040 is evaluating the safety and efficacy of the combination in patients with previously treated advanced HCC. Primary endpoints include safety and tolerability, and objective response rate based on investigator assessment using Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1).
Secondary endpoints include disease control rate, duration of response, overall survival, time to response, time to progression and progression-free survival.
Liver cancer is the fourth most frequent cause of cancer death worldwide and HCC, the most common type of liver cancer, is the fastest rising cause of cancer-related death in the United States.
Although the FDA has approved several new agents in the second-line setting in recent years, HCC is often diagnosed in the advanced stage and has a poor prognosis, underscoring the need for new treatment options.
While most cases of HCC are caused by hepatitis B virus or hepatitis C virus infections, metabolic syndrome and nonalcoholic steatohepatitis are rising in prevalence and expected to contribute to increased rates of HCC.
Opdivo is a programmed death-1 immune checkpoint inhibitor that is designed to uniquely harness the body's own immune system to help restore anti-tumor immune response.
By harnessing the body's own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo's leading global development program is based on Bristol-Myers Squibb's scientific expertise in the field of Immuno-Oncology, and includes a range of clinical trials across all phases, including Phase 3, in a variety of tumor types.
To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. 
Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China.
In October 2015, the company's Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.