On March 1, 2018 FLAG Therapeutics Inc. reported that the U.S. Food and Drug Administration’s Office of Orphan Products Development and the European Medicines Agency (EMA) have both granted Orphan Drug Designation to FLAG-003 for the treatment of glioma (Press release, Flag Therapeutics, MAR 1, 2016, View Source [SID1234525570]). Gliomas (including Glioblastoma) are the most aggressive forms of brain cancer and carries a very poor prognosis for survival and is one of the deadliest forms of cancer. Two and 5-year survival rates are 27% and 10%, with the median progression-free survival (PFS) being only 6.9 months.

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Orphan status is granted by the FDA to promote the development of products that demonstrate promise for the treatment of rare diseases – those which affect fewer than 200,000 Americans annually. Orphan drug designation entitles FLAG Therapeutics to 7 years marketing exclusivity following product launch in the United States (10 years marketing exclusivity in the EU) and enables the company to apply for research funding, tax credits, a waiver from FDA (PDUFA) user fees, FDA assistance in clinical trial design, and access to the central authorization procedure within the European Union.

FLAG-003 for the treatment of Glioma

FLAG-003 is a small molecule which exerts both cytotoxic and cytostatic activity due to two distinct and well characterized mechanisms of action. It possesses cytotoxic anti-tubulin activity by binding to the colchicine site of tubulin causing microtubule depolymerization. It also possesses anti-angiogenic activity through binding and inhibition of RTK receptor tyrosine kinase (RTKs) activity. The anti-tubulin and anti-angiogenic activities of FLAG-003 have translated into potent antitumor and anti-vascular effects in vivo with significantly better inhibitory activity on GBM tumor growth and vascularization than the currently approved chemotherapy, temozolomide (TMZ).