ArQule Says FDA Grants Rare Pediatric Disease Designation To Miransertib

(RTTNews) - ArQule Inc. (ARQL) said that the U.S. Food and Drug Administration has granted Rare Pediatric Disease Designation to miransertib (ARQ 092) for the treatment of Proteus syndrome. Under the FDA's rare pediatric disease priority review voucher program, the sponsor may be eligible for a voucher that can be used to obtain priority review for a subsequent human drug application if it meets relevant statutory requirements associated with the program, including FDA approval of the drug in this indication.

The FDA previously granted Orphan Drug Designation to miransertib for the treatment of Proteus syndrome, a rare disease characterized by overgrowth of the skeleton, skin, adipose tissue and central nervous system. Miransertib is an orally available, selective pan-AKT inhibitor.

ArQule is enrolling a phase 1/2 trial for Overgrowth Diseases, including Proteus syndrome and PROS, driven by either the AKT or PI3K mutation. The phase 1 portion of the trial is enrolling six patients in a dose escalation cohort. An additional 10 patients will be enrolled in an expansion cohort as part of the phase 2 portion of the trial.

In parallel with the company sponsored phase 1/2 trial, ArQule will continue to provide miransertib on a named patient basis to those patients unable to travel to a clinical trial site due to severe disease.