FDA grants orphan drug designation to durvalumab-tremelimumab combination for liver cancer
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The FDA granted orphan drug designation to durvalumab and tremelimumab for treatment of patients with advanced hepatocellular carcinoma.
Durvalumab (Imfinzi, AstraZeneca) is a human monoclonal antibody that binds to PD-L1. It inhibits interaction of PD-L1 with PD-1 and CD80. Tremelimumab (MedImmune/AstraZeneca) is an investigational anti-CTLA-4 monoclonal antibody.
The ongoing phase 3 HIMALAYA trial is designed to evaluate the combination of durvalumab and tremelimumab for patients with advanced and unresectable HCC who are ineligible for localized treatment and have not received prior systemic therapies.
The HIMALAYA trial will be the first to evaluate therapy with two immune checkpoint inhibitors as first-line treatment for advanced HCC, according to AstraZeneca.
“Many patients with liver cancer are diagnosed and treated only after the disease is advanced, and there is an urgent need for new effective and tolerable treatments,” José Baselga, MD, PhD, executive vice president of oncology research and development at AstraZeneca, said in a company-issued press release. “We are eager to bring new potential options to these patients and look forward to the results of our ongoing phase 3 HIMALAYA trial later this year.”
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.