Glycomine, Inc., a biotechnology company specializing in treatments for rare diseases, has announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to GLM101, a mannose-1-phosphate replacement therapy being developed to treat phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG). “Fast Track Designation for GLM101 emphasizes its potential to address the significant unmet medical need in PMM2-CDG patients,” said Dr. Rose Marino, Chief Medical Officer of Glycomine. “As we continue to enroll pediatric patients in our Phase 2 clinical study, we are optimistic about the promising clinical benefits we are observing with GLM101. We eagerly anticipate advancing its development under the Fast Track framework.”
The Phase 2 clinical study, GLM101-002, has enrolled 10 adult and five adolescent patients with PMM2-CDG in the U.S. and Spain (ClinicalTrials.gov Identifier: NCT05549219). Study participants were treated with GLM101 at doses of 10 mg/kg (n=3), 20 mg/kg (n=3), or 30 mg/kg (n=9) for up to 24 weeks. Over 350 doses have been administered, and the therapy has been well tolerated with no severe adverse events reported—only mild to moderate side effects observed. The trial is expected to continue with pediatric patients aged 2 years and older.
The FDA’s Fast Track program is designed to accelerate the development and review of new treatments for serious conditions that address significant unmet medical needs. Companies with Fast Track Designation benefit from more frequent communication with the FDA and may be eligible for accelerated approval or priority review if certain conditions are met.
As Steve Jobs once said, “Innovation distinguishes between a leader and a follower.” Glycomine’s advancements in treating rare conditions reflect this spirit of innovation, as they forge ahead in the battle against PMM2-CDG.
About GLM101
GLM101 is a mannose-1-phosphate replacement therapy in development to treat phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), previously known as CDG Type Ia. GLM101 has received Orphan Drug Designation in the U.S. and Europe and Rare Pediatric Disease Designation in the U.S. PMM2-CDG is caused by genetic mutations that lead to a deficiency of the enzyme phosphomannomutase 2 (encoded by the PMM2 gene). GLM101 is designed to deliver mannose-1-phosphate directly into cells and thereby bypass the PMM2 enzyme deficiency and address disease-causing PMM2 mutations to restore pathway function.
About Glycomine, Inc.
Glycomine is a clinical-stage biotechnology company developing novel drugs for serious rare disorders of metabolism and protein misfolding for which no other therapeutic options exist. The company’s approach is to use replacement therapies – substrates, enzymes, or proteins – and to target those molecules to clinically relevant cellular compartments.
