Keywords: Verastem. Defactinib, Orphan designation, USA, Mesothelioma
Article | 25 July 2013
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.
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“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.”
Verastem recently outlined details of the registration-directed clinical study of defactinib in patients with malignant pleural mesothelioma. This study is designed as a double-blind, placebo-controlled trial with an expected enrollment of around 350-400 patients at clinical sites in 11 countries.
In discussions with regulatory agencies worldwide
“We are in discussions with the regulatory agencies and clinical investigators worldwide,” said Joanna Horobin, Verastem’s chief medical Officer. “We recently held our investigator meetings for the physicians conducting the trial in the USA and Australia and we are on track to begin enrolling patients in the third quarter. We plan to open sites worldwide on a rolling basis as we clear regulatory and clinical review in each country,” she added.
“Cancer stem cells play a central role in treatment resistance in many types of cancers,” noted Christoph Westphal, Verastem’s executive chairman. “We believe new treatment options targeting cancer stem cells will be critical to achieve a durable clinical benefit for patients. This designation will provide us with many benefits as we pursue the development of defactinib for the treatment of mesothelioma,” he said.
In addition to mesothelioma, Verastem recently announced the completion of the Phase I stage and initial data from an ongoing Phase I/Ib study of defactinib in combination with weekly paclitaxel for patients with ovarian cancer (The Pharma Letter July 24). Verastem also expects to initiate a Phase I study in Japan, and a Phase II trial in KRAS-mutated non-small cell lung cancer, for defactinib during the third quarter of 2013.
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