Verastem, Inc., a clinical-stage biopharmaceutical company, has filed an application seeking orphan drug designation in the US and the EU for the use of VS-6063 in mesothelioma. VS-6063 is anticipated to enter a potentially pivotal study in mesothelioma midyear 2013.

VS-6063 is an oral small molecule inhibitor of focal adhesion kinase (FAK), which is a critical pathway for cancer stem cells. VS-6063 was well-tolerated and demonstrated signs of clinical activity in a phase 1 study in advanced solid tumors. VS-6063 is currently in a phase 1/2 study in patients with advanced ovarian cancer, where VS-6063 is being tested in combination with paclitaxel. VS-6063 is anticipated to enter a potentially pivotal study in mesothelioma midyear 2013.

Orphan designation grants US market exclusivity to a drug for the treatment of a specified condition for a period of seven years following FDA marketing approval and 10 years of market exclusivity in member states of the EU if received. Additional potential benefits of orphan designation include development grants, tax credits related to clinical trial expenses, protocol development assistance and exemption or reduction from certain regulatory fees.

Application for orphan drug designation is available for treatment of medical conditions affecting fewer than 200,000 people in the US, or no more than 5 in 10,000 people in the EU, from the respective regulatory agencies in each region.

Verastem, Inc. is a clinical-stage biopharmaceutical company focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells.