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Verastem’s Newest Drug Receives Orphan Drug Status

Verastem, an innovative biopharmaceutical company that focuses its research and development on developing drugs that target cancer stem cells, has announced that one of their latest drugs in development, VS-5584, has received orphan drug designation from the European Commission. An orphan drug status is a special designation that is reserved specifically for medications and biological products that are developed for the treatment of a rare disease or condition. The status is generally requested by the drug’s sponsor

Though an orphan drug is still required to go through all of the same regulatory steps as those without the designation, the manufacturer and sponsor gets certain benefits from the designation. These may include tax credits for the cost of clinical testing and waivers of fees. Orphan drug designation is available in the United States as well as from the European Commission, and is essential to encourage drug developers to work on medications for illnesses that strike so few people that the companies have no financial incentive to invest in research.

Verastem’s VS-5584 is a dual mTORC1/2 and PI3K inhibitor that just began dosing patients in their first week of Phase 1 clinical trials. The patient being treated has advanced solid mesothelioma tumors. The clinical trial hopes to attract almost sixty patients with relapsed malignant pleural mesothelioma, which will be treated with a combination of the VS-5584 and the company’s other mesothelioma drug, VS-6063.

According to Robert Forrester, President and Chief Executive Officer of Verastem, “Following diagnosis, there are very few treatment options for mesothelioma patients. This European orphan designation could help facilitate the regulatory process for development of VS-5584, and if our clinical studies are successful, may allow us to bring this potential new treatment option to patients more rapidly. We have dosed the first patient in our first in-human combination study of VS-5584 and VS-6063 in mesothelioma and we look forward to providing updates at the trial progresses.”

The orphan drug designation from the European Commission gives drug developers the additional financial benefit of up to ten years of market exclusivity for their product if it is approved for market in the European Union and they are able to maintain their designation.

Terri Oppenheimer

Terri Oppenheimer is an experienced blog writer, editor, and proofreader. She graduated from the College of William and Mary with a degree in English. She specializes in providing content for websites and finds tremendous enjoyment in the things she learns while doing her research. Her specific areas of expertise include health, medical research, and law.

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