FDA Grants AstraZeneca’s Tremelimumab Orphan Drug Status for Mesothelioma

Illustration of mesothelioma research

The U.K.-based pharmaceutical company AstraZeneca announced on Wednesday that the U.S. Food & Drug Administration (FDA) has granted tremelimumab orphan drug status for the treatment of malignant mesothelioma.

In the company’s press release, Robert Iannone, Head of Immuno-oncology at AstraZeneca’s Global Medicines Development division, said that there is a crucial need to develop additional mesothelioma treatments, since less than 5% of the deadly cancer’s victims live more than five years. “Our aim is to rapidly advance the development of tremelimumab,” Iannone stated, adding that the company hoped the drug would become a viable new option for treatment of the disease.

Tremelimumab Details

The drug tremelimumab is an anti-CTLA-4 monoclonal antibody, which means that it blocks production of an inhibitor that destroys Cytotoxic T lymphocytes (CTLs) – a type of white blood cell produced naturally by the body that can recognize and kill cancer. With the inhibitor blocked, the body can produce potentially cancer-defeating CTLA-4 antibodies more quickly. A clinical study in 2012 was established to study the effects of the drug on malignant mesothelioma.

Another version of anti-CTLA-4 monoclonal antibody, known as ipilimumab (brand name Yervoy), has been tested in treatments for various forms of cancer, including melanoma, prostate cancer, and lung cancer.

Orphan Drug Status

According to the Orphan Drug Act of January 1983, an orphan drug is any drug that treats disorders affecting less than 200,000 people in the U.S. Designation of orphan drug status gives the pharmaceutical company exclusive rights to sell the drug without competition for seven years. Companies also may receive certain tax incentives for clinical trials involving orphan drugs.

AstraZeneca is also testing tremelimumab for use in fighting certain forms of lung cancer, as well as head and neck cancer. If these uses are found to be effective and are approved, the drug could become much more widely available.