BioTuesdays

Applied Therapeutics AT-007 gets FDA rare pediatric disease and orphan drug designations

The FDA granted Applied Therapeutics’ (NASDAQ:APLT) AT-007 rare pediatric disease and orphan drug designations for the treatment of PMM2-congenital disorder of glycosylation (PMM2-CDG).

PMM2-CDG is a rare disease caused by a deficiency in phosphomannomutase-2, an enzyme required for systemic glycosylation of proteins. The disease causes multiple organ failure and severe disability, with a mortality rate of 20% in the first four years of life. 

The company plans to initiate a clinical study of AT-007, an aldose reductase inhibitor, in 2021. 

“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,” Dr. Riccardo Perfetti, Applied Therapeutics’ CMO, said in a statement.

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