PharmaTher Submits Orphan Drug and Rare Pediatric Disease Designation Applications to FDA for 3D-Printed Creatine Monohydrate in GAMT Deficiency
HighlightsTwo FDA designation applications submitted for creatine monohydrate in guanidinoacetate methyltransferase (GAMT) deficiency: Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPD).First-in-class product opportunity. No FDA-approved treatment exists for GAMT deficiency. Families measure and mix high-dose creatine powder by hand, several times a day, for life.3D printing for children. Weight-banded, taste-masked, 3D-printed dissolvable tablets give each child a...