US orphan designation for defactinib in mesothelioma

US cancer drug developer Verastem (Nasdaq: VSTM) says that its lead cancer stem cell inhibitor, VS-6063 (defactinib), has received orphan drug designation from the US Food and Drug Administration for use in the treatment of mesothelioma, a rare form of lung cancer.

The designation is designed to encourage the development of drugs which may provide significant benefit to patients suffering from rare diseases. The designation provides eligibility for a seven-year period of market exclusivity in the USA after product approval, FDA assistance in clinical trial design, and an exemption from FDA user fees.

“Mesothelioma is among the most aggressive and lethal cancers but has limited treatment options,” said Robert Forrester, Verastem’s president and chief executive, adding: “We are pleased that the FDA recognizes the significant unmet medical need in mesothelioma. We previously received orphan medicinal product status for defactinib in Europe and these two designations are an important component of our development strategy.”