As reported in the Wall Street Journal online Verastem Inc. has been granted orphan drug status from the European Medicines Agency (EMA) for the use of VS-6063 as an investigational treatment for mesothelioma. Mesothelioma, an aggressive and rare cancer for which there is no cure, is caused by an asbestos exposure. Mesothelioma typically affects the lining of the lungs but can also affect the lining of the abdomen and the lining of the heart. Treatment options are limited and sadly, most patients succumb to the illness within a short time of their diagnosis. The need for new treatments or a cure is critical to help save the lives of the individuals suffering from this deadly disease. Verastem Inc., which is focused on developing drugs to treat cancer by targeting of cancer stem cells, is also seeking orphan status for the treatment–known as VS-6063–from the U.S. Food and Drug Administration (FDA).
Verastem Inc., which is located in Cambridge, Massachusetts, received their orphan drug designation from the EMA under a very similar program to that of the FDA with comparable legislation. Established by the Orphan Drug Act of 1983, the FDA’s Orphan Drug Designation (ODD) Program is intended to promote the study, development and approval of drugs that would benefit individuals suffering from uncommon, rare illnesses. Because many drug companies are reluctant to finance the research for diseases that do not affect a great number of individuals and would not generate significant revenue for their companies, the Program seeks to encourage pharmaceutical companies to develop drugs that could be used in the prevention, diagnosis and treatment of rare diseases and disorders, specifically those conditions that affect less than 200,000 people in the United States.
FDA orphan drug designation provides companies benefits such as tax credits or reductions, no FDA approval application fees, the assistance of the Office of Orphan Product Development throughout the process, and a 7-year marketing exclusivity. The EMA’s orphan drug designation is applicable to a broader range of conditions, including some tropical diseases, and marketing exclusivity is 10 years.
The oral drug, VS-6063, is currently being investigated for the treatment of ovarian cancer and will be the center of a mesothelioma study later this summer. The study will include an estimated 350-to 400-mesothelioma patients in several different countries including the United States, Europe, South Africa, New Zealand, Australia and Canada. The drug is considered to be an inhibitor of focal adhesion kinase (FAK). Verastem has indicated that a protein substance, merlin, as well as the FAK have been found to be inactive in patients suffering from mesothelioma. This inactivity allows the mesothelioma cells to grow and spread. VS-6063 will aim to target the cancer cells through the inhibition of the FAK signaling pathway.
If you or someone you love has been diagnosed with mesothelioma, lung cancer, asbestosis or another asbestos-related disease, contact the Philadelphia mesothelioma attorneys at the law firm of Brookman, Rosenberg, Brown & Sandler. Our law offices, conveniently located in Center City Philadelphia, are easily accessible from all communities in Pennsylvania and New Jersey; however, we will come to you if that is more convenient. Call us today at 1-800-369-0899 to schedule a free consultation or contact us online.