APLT - Applied Therapeutics receives Rare Disease Orphan designations for AT-007

The FDA grants Pediatric Rare Disease and Orphan Designation to Applied Therapeutic's (APLT) +3.7% AT-007 for treatment of PMM2-CDG, a disease caused by deficiency in the critical enzyme phosphomannomutase-2, required for systemic glycosylation of proteins.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. 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.Source: Press Release

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Applied Therapeutics receives Rare Disease, Orphan designations for AT-007