FDA授予durvalumab-tremelimumab联合治疗肝癌的孤儿药称号

StephenW

FDA News
Immuno-Oncology Resource Center
FDA grants orphan drug designation to durvalumab-tremelimumab combination for liver cancer
January 21, 2020
Photo of Jose Baselga
José Baselga, MD, PhD

The FDA granted orphan drug designation to the combination of durvalumab plus 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 plus 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.

StephenW

FDA新闻
免疫肿瘤学资源中心
FDA授予durvalumab-tremelimumab联合治疗肝癌的孤儿药称号
2020年1月21日
Jose Baselga的照片
医学博士JoséBaselga

FDA授予durvalumab加tremelimumab组合的孤儿药治疗晚期肝细胞癌患者。

Durvalumab（Imfinzi，AstraZeneca）是与PD-L1结合的人单克隆抗体。它抑制PD-L1与PD-1和CD80的相互作用。 Tremelimumab（MedImmune / AstraZeneca）是研究性的抗CTLA-4单克隆抗体。

正在进行的HIMALAYA 3期临床试验旨在评估durvalumab与tremelimumab的联合治疗，这些患者不适合进行局部治疗且尚未接受过先前的全身疗法，用于晚期和不可切除的HCC患者。

据阿斯利康公司称，HIMALAYA试验将是第一个评估使用两种免疫检查点抑制剂作为晚期HCC的一线治疗方法的试验。

阿斯利康肿瘤学研究与发展执行副总裁何塞·巴塞尔加（JoséBaselga）医师表示：“许多肝癌患者只有在疾病发展后才能得到诊断和治疗，因此迫切需要新的有效且可耐受的治疗方法。”在公司发布的新闻稿中。 “我们渴望为这些患者带来新的潜在选择，并期待我们今年晚些时候正在进行的三期HIMALAYA试验的结果。”

FDA孤儿产品开发办公室将孤儿药指定给旨在安全有效地治疗，诊断或预防在美国影响不到20万人的罕见疾病或病症的新型药物和生物制剂。该名称使制造商有资格获得各种激励措施，包括针对合格的临床试验的税收抵免，以及（经监管部门批准）7年的市场专有权。