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Verastem Receives Orphan Drug Status from FDA

Less than a month after the European market provided its orphan drug designation for Verastem’s VS-5584 drug for the treatment of mesothelioma, the United States’ Food and Drug Administration is following suit. Orphan drug status is a special designation provided to drugs that are created for the specific purpose of treating a rare condition. The idea behind the designation is that it provides drug companies and researchers with an incentive to continue their work in creating medications that would not otherwise be financially worthwhile because of the small number of people that would benefit from them. VS-5584 is specifically designed for the treatment of mesothelioma, a rare and deadly form of cancer that only impacts about 3,000 patients per year in the United States.

Verastem is a U.S. manufacturer that specializes in finding ways to use stem cells to fight cancer. Their VS-5584 is focused on inhibiting the cancer stem cell signaling pathways. It does this by identifying and targeting class 1 P13K enzymes. These enzymes act to advance cell growth and survival. The drug also targets a pair of protein complexes called mTORC1 and mTORC2 which control protein synthesis and cellular metabolism. The drug is currently being studied in two different clinical trials. The first is a Phase I trial being conducted on mesothelioma patients in the United States as well as in the United Kingdom. It is assessing how VS-5584 works when combined with defactinib (VS-6063), another experimental medication made by Verastem and also being independently evaluated in a separate clinical trial. The second is also a Phase I trial that is aimed at determining whether VS-5584 is effective in the treatment of advanced solid tumors.

When the U.S. Food and Drug Administration provides orphan drug designation, it translates into a number of different incentives, including market exclusivity and tax credits. Speaking to the importance of the designation, Verastem’s Chief Executive Officer Robert Forrester said, “This is an important regulatory milestone for Verastem and, together with our European orphan medicinal product designation, will facilitate our global development of VS-5584 to help improve the available treatment options for patients suffering from this highly aggressive cancer.” He also said that the company will be “taking full advantage of the opportunities that orphan designation allows in order to bring this potential new treatment option to patients as rapidly as possible.”

Terri Oppenheimer

Terri Oppenheimer is an independent writer, editor and proofreader. She graduated from the College of William and Mary with a degree in English. Her dreams of a writing career were diverted by a need to pay her bills. She spent a few years providing copy for a major retailer, then landed a lucrative career in advertising sales. With college bills for all three of her kids paid, she left corporate America for a return to her original goal of writing. She specializes in providing content for websites and finds tremendous enjoyment in the things she learns while doing her research. Her specific areas of interest include health and fitness, medical research, and the law.

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