Posted by Michael Wonder on 24 Sep 2020
FDA grants AT-007 paediatric rare disease designation and orphan designation for treatment of PMM2-CDG
24 September 2020 - Applied Therapeutics announced today that the U.S. FDA has granted AT-007 both paediatric 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.
