FDA Grants AT-007 Pediatric Rare Disease Designation and Orphan Designation for Treatment of PMM2-CDG

Applied Therapeutics announces today that the U.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 Therapeutics plans to initiate a clinical study in 2021 and is currently working with CDG experts and the FDA to design a robust clinical program.

Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics Said: 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, 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.

Source: Applied Therapeutics

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