New mesothelioma drug granted orphan status from EU

26 Jun 2013 by under News, Research/Treatment

Verastem banner 100x100 New mesothelioma drug granted orphan status from EUDrug maker Verastem Inc. has obtained approval from the European Commission to proceed with the development of a potential new mesothelioma treatment, which has been granted orphan status.

Orphan Medicinal Product Designation provides up to 10 years of potential market exclusivity if the drug is approved for marketing in Europe. The status also gives Verastem enhanced interaction with the European drug regulating agency in developing trials and preparing the drug for final approval.

Mesothelioma is a form of cancer resulting from exposure. It primarily affects the lining of the lungs or the abdomen, but may also rarely affect the lining of the heart. There is currently no known cure for mesothelioma.

The drug candidate is known as . Clinical trials on the new treatment are scheduled to begin this summer. VS-6063 also is being studied in a Phase 1/1B trial as a treatment for ovarian cancer, in combination with another already approved cancer drug.

Dr. Joanna Horobin, Verastem chief medical officer, issued a statement saying “Mesothelioma is a devastating disease with limited treatment options. We are working with investigators throughout Europe and internationally to bring a new treatment option for these patients.”

Verastem, on its website, says VS-6063 is an “orally available, small molecule inhibitor of focal adhesion kinase (FAK).” The drug maker says a biomarker test will be used to identify mesothelioma patients who are low in a marker known as Merlin. They note their research indicates Merlin-low mesothelioma cells and tumors may be sensitive to this FAK inhibition. Versastem estimates 40-50 percent of mesothelioma patients lack Merlin.

Verastem is a clinical-stage biopharmaceutical company based in Cambridge, Mass. Its mission is to discover and develop drugs to treat cancer by the targeted killing of cancer stem cells.

Sources:

Boston Business Journal
Verastem

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