GLM101 is designed to deliver mannose-1-phosphate into cells and thereby bypass disease-causing PMM2 mutations to restore pathway function. GLM101 has received Orphan Drug Designation in the U.S. and E.U., as well as Rare Pediatric Disease Designation, Fast Track Designation, and Breakthrough ... GLM101 is designed to deliver mannose-1-phosphate into cells and thereby bypass disease-causing PMM2 mutations to restore pathway function. GLM101 has received Orphan Drug Designation in the U.S. and E.U., as well as Rare Pediatric Disease Designation, Fast Track Designation, and Breakthrough Therapy Designation in the U.S. SAN CARLOS, Calif.--(BUSINESS WIRE)--#CDG--Glycomine, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to GLM101 for the treatment of phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG), a serious, multisystem disorder with no approved treatments. FDA grants Breakthrough Therapy designation to investigational drugs for serious conditions when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint.