Regulatory Affairs

FDA grants priority review to Merck’s sBLA for Keytruda to treat advanced hepatocellular carcinoma

Published 12 July 2018

Merck announced that the US Food and Drug Administration (FDA) has accepted and granted priority review for a new supplemental biologics license application (sBLA) seeking approval for anti-PD-1 therapy, Keytruda, as a treatment for previously treated patients with advanced hepatocellular carcinoma (HCC).

This sBLA, which is seeking accelerated approval for this new indication, is based on data from the Phase 2 KEYNOTE-224 trial, which were recently presented at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting and published simultaneously in The Lancet Oncology.

The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of Nov. 9, 2018.

“There continues to be a significant need for new options in the treatment of advanced hepatocellular carcinoma, which is the most common type of liver cancer,” said Dr. Scot Ebbinghaus, vice president, clinical research, Merck Research Laboratories.

 “The data supporting our application provide a clear rationale for the advancement of the KEYTRUDA clinical program for hepatocellular carcinoma, and we are grateful for the opportunity to work with the FDA to potentially bring KEYTRUDA to patients living with this difficult-to-treat cancer.”

Merck’s broad immuno-oncology clinical development program includes more than 10 Merck-sponsored studies investigating KEYTRUDA in gastrointestinal cancers, including HCC.

In addition to KEYNOTE-224, Merck has two ongoing pivotal Phase 3 studies, KEYNOTE-240 and KEYNOTE-394, evaluating KEYTRUDA as monotherapy in second-line HCC, as well as ongoing trials investigating KEYTRUDA in combination with other treatments including LENVIMA.

 Hepatocellular carcinoma (HCC) is the most common type of liver cancer in adults. In the U.S., the incidence of liver cancer has more than tripled since 1980, and it is estimated that 42,200 new cases of liver cancer will be diagnosed this year.

Risk factors for liver cancer include gender, ethnicity, chronic viral hepatitis (Hep-B or Hep-C) infection, cirrhosis, alcohol use and metabolic syndrome. Hepatocellular carcinoma – which is frequently diagnosed at an advanced stage – has one of the highest mortality rates of solid cancers, with a five-year survival rate of less than 15 percent.

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industry’s largest immuno-oncology clinical research program, which currently involves more than 750 trials studying KEYTRUDA across a wide variety of cancers and treatment settings.

The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient’s likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Source: Company Press Release