ArQule announces FDA Fast Track Designation for rare disease med

ArQule announced that the FDA has granted Fast Track Designation to miransertib for the treatment of PIK3CA-Related Overgrowth Spectrum, or PROS, a group of related, ultra-rare genetic disorders characterized by excessive tissue growth in various parts of the body. The FDA’s Fast Track program aims to expedite the development and review of drugs which treat serious or life-threatening conditions and have demonstrated the potential to address unmet clinical needs. Miransertib has already been granted Rare Pediatric Disease Designation by the FDA and Orphan Designation by the FDA and European Medicines Agency in the rare overgrowth disease, Proteus syndrome. During the past three years, miransertib has been tested in a Phase 1 NIH/NHGRI-sponsored trial in Proteus Syndrome. More recently, we have been conducting a Phase 1/2 company-sponsored trial in PROS and a single patient use program with select physicians.
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