The Food and Drug Administration (FDA) has granted Fast Track Designation to miransertib (ArQule) for the treatment of PIK3CA-related overgrowth spectrum (PROS), a group of ultra-rare genetic disorders characterized by excessive tissue growth in various parts of the body.

Miransertib is an orally available, selective, pan-AKT (protein kinase B) inhibitor that inhibits AKT1, 2 and 3 isoforms. Currently, there are 3 ongoing studies of miransertib: a Phase 1/2 study for PIK3KA-related overgrowth spectrum (PROS), a Phase 1 study for ultra-rare Proteus syndrome, and a Phase 1b study in combination with anastrozole, in patients with advanced endometrial cancer with AKT and PI3K mutations.

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Miransertib has already been granted Rare Pediatric Disease Designation and Orphan Drug Designation by the FDA for Proteus syndrome. Dr. Andrea Bartuli, Director of the Genetic and Rare Disease Unit of Bambino Gesù Pediatric Hospital and the principal investigator of the PROS trial said, “I am committed to helping advance miransertib as the potential first effective therapeutic for patients with PROS.”

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