Analysis Group Researchers Support FDA Breakthrough Therapy Designation for Drug Candidate in Rare Genetic Disease

October 15, 2026

Phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG) is a rare, inherited metabolic disorder. It presents in infancy and persists into adulthood with severe multi-system disease and up to 20 percent premature mortality in the early years of life. No treatments are currently approved for the disease. Given improvements in ataxia and other key measures among patients with PMM2-CDG studied after 24 weeks of Glycomine’s investigational GLM101 treatment, the US Food and Drug Administration (FDA) has granted it Breakthrough Therapy designation.

To contextualize clinical outcomes, an Analysis Group team – led by Managing Principal James Signorovitch, Vice Presidents Jess Marden and Cameron Fowler, Manager Andres Gomez-Lievano, and Associate Emma Billmyer – analyzed Phase 2a data relative to the natural history of patients with untreated PMM2-CDG. Glycomine is continuing to evaluate GLM101 in its global randomized Phase 2b POLAR study.

Read Glycomine's press release