Benzinga | Sep 24, 2020 08:08AM EDT

Applied Therapeutics Announces FDA Grants AT-007 Pediatric Rare Disease Designation And Orphan Designation For Treatment Of PMM2-CDG

NEW YORK, Sept. 24, 2020 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (NASDAQ:APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, announced todaythat theU.S. Food and Drug Administration(FDA) has granted AT-007 both Pediatric Rare Disease Designation and Orphan Drug Designation for treatment of PMM2-CDG. PMM2-CDG is a debilitating rare disease caused by deficiency in the critical enzyme phosphomannomutase-2, required for systemic glycosylation of proteins. PMM2-CDG causes multiple organ failure and severe disability, resulting in approximately 20% mortality in the first four years of life. There are currently no drugs approved to treat PMM2-CDG.



AT-007, a novel CNS penetrant Aldose Reductase inhibitor (recently given the INN scientific name gavorestat) has demonstrated biological activity in a validated model of PMM2-CDG. Applied Therapeuticsplans to initiate a clinical study in 2021 and is currently working with CDG experts and the FDA to design a robust clinical program.

"PMM2-CDG is a debilitating disease with no drugs available, which is central to Applied Therapeutics' mission of creating life-changing treatments for patients who desperately need them," said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. "Over the past year we have been working closely with clinicians, researchers and families of PMM2-CDG patients, and are grateful for their collaboration and support. We are pleased that the FDA recognizes the transformative potential of AT-007 in treating patients with PMM2-CDG."