SAN CARLOS, Calif.– Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for GLM101, a mannose-1-phosphate replacement therapy in development for the treatment of patients with phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG).
“Fast Track Designation for GLM101 highlights its potential to meet the serious unmet medical need of patients with PMM2-CDG,” said Rose Marino, M.D., Chief Medical Officer of Glycomine. “As we continue to enroll pediatric patients in our Phase 2 clinical study, we are encouraged by our initial data showing promising evidence of clinical benefit with GLM101. We look forward to progressing development of GLM101 within the Fast Track framework.”
The GLM101-002 Phase 2 clinical study has enrolled 10 adult and five adolescent patients with PMM2-CDG in the U.S. and Spain (ClinicalTrials.gov Identifier: NCT05549219). Study participants have completed treatment with GLM101 at either 10 mg/kg (n=3), 20 mg/kg (n=3), or 30 mg/kg (n=9) for up to 24 weeks. More than 350 doses of GLM101 have been administered to PMM2-CDG patients. The drug appears to be well tolerated with no serious adverse events, and only mild to moderate adverse events reported to date. The study is planned to continue enrollment in pediatric patients ≥ 2 years of age.
The FDA’s Fast Track program is designed to facilitate the development and expedite the review of novel potential therapies intended to treat serious conditions and address significant unmet medical needs. Companies whose programs are granted Fast Track designation are eligible for more frequent interactions with the FDA during clinical development and potentially accelerated approval and/or priority review, if relevant criteria are met.
