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AstraZeneca’s cancer drug tremelimumab obtains FDA orphan drug status

The US Food and Drug Administration has granted orphan drug designation for AstraZeneca's anti-CTLA-4 monoclonal antibody, tremelimumab, to treat patients with malignant mesothelioma, a rare type of cancer.

AstraZeneca Headquarters London

Tremelimumab is one of a number of immuno-oncology products being developed by AstraZeneca and MedImmune to harness the body’s own immune system to fight cancer.

The drug stimulates the immune system to destroy cancer cells through binding to the protein CTLA-4, expressed on the surface of activated T-lymphocytes.

AstraZeneca senior vice-president, head of Immuno-oncology, Global Medicines Development Robert Iannone said: "There is a significant need for new treatment options for patients with mesothelioma because fewer than five percent of patients currently survive beyond five years, even when they receive timely diagnosis and care.

"Our aim is to rapidly advance the development of tremelimumab as a potential new treatment option for these patients."

Currently, tremelimumab is also being evaluated in combination with AstraZeneca’s anti PD-L1 investigational immunotherapy, MEDI4736, in tumor types including non-small cell lung cancer (NSCLC) and head and neck cancer.

The drug is also being studied in combination with Iressa (gefitinib) in EGFR mutated non-small cell lung cancer and with MEDI6469 in solid tumors.

AstraZeneca’s broad pipeline of next-generation medicines is focused on four main disease areas – ovarian, lung, breast and haematological cancers.


Image: AstraZeneca headquarters in London, UK. Photo: courtesy of AstraZeneca plc.