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AstraZeneca gains FDA orphan status for new cancer drug

Gains a longer patent life for the investigational lung disease drug

AZ HQ London 

The FDA has granted AstraZeneca an orphan drug designation for its anti-CTLA-4 monoclonal antibody, tremelimumab, for the treatment of malignant mesothelioma.

Mesothelioma is a rare, aggressive cancer that most often affects the lining of the lungs and abdomen and typically comes as a result of contact with asbestos.

Available treatments for mesothelioma are very limited, particularly for patients with advanced disease.

This designation provides orphan status to drugs and biologics, which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. 

It also allows the drug an extended patent life - an incentive aimed at encouraging pharma to create drugs for these types of rare disorders.

Robert Iannone, senior VP and head of immuno-oncology, global medicines development at AstraZeneca, said: “There is a significant need for new treatment options for patients with mesothelioma because fewer than 5% 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.”

Tremelimumab is part of the broad pipeline of immuno-oncology assets being developed by AstraZeneca and its biologics research and development arm, MedImmune, which are designed to harness the body's own immune system to fight cancer. 

It is a fully human monoclonal antibody, which stimulates the immune system to destroy cancer cells through binding to the protein CTLA-4, expressed on the surface of activated T-lymphocytes.

In addition to being investigated as a monotherapy treatment for patients with mesothelioma, tremelimumab is currently being studied in combination with AstraZeneca's anti PD-L1 investigational immunotherapy, MEDI4736, in tumour types including non-small cell lung cancer and head and neck cancer. AZ is developing this drug in the late stage of testing, and is hoping to gain access to the PD-1 market - currently dominated by BMS and Merck & Co - and the $30bn analysts believe it could be worth in the next decade. 

It is also being studied in combination with AstraZeneca's established oncology treatment Iressa (gefitinib) in EGFR mutated non-small cell lung cancer and with MEDI6469 (a murine OX40 agonist) in solid tumours.

Article by
Ben Adams

16th April 2015

From: Regulatory



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