Verastem have announced that their mesothelioma candidate VS-6063 has received Orphan Medicinal Product Designation from the European Commission. VS-6063 is a small molecule inhibitor of focal adhesion kinase (FAK), which has a critical role for cancer stem cell survival and disease progression. Studies have shown that mesothelioma cells and tumours low in a marker called Merlin are particularly sensitive to FAK inhibitors.
Cancer stem cells (CSCs) are thought to be an underlying cause of tumour recurrence and metastasis, and several biopharmaceutical companies are developing therapies to target them. Very recently, Stemline announced that their CSC-targeting therapy SL-401 had been granted orphan drug designation by the FDA for the treatment of blastic plasmacytoid dendritic cell neoplasm.
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If you want to know more about innovation and strategy in the orphan drug market, you might be interested in attending the World Orphan Drug Congress Asia, 18-19 June 2013, Singapore, or the 4th World Orphan Drug Congress Europe, in Geneva on the 14th November 2013.