The Food and Drug Administration (FDA) has granted Orphan Drug designation to MZE001 for the treatment of Pompe disease.

Pompe disease is a rare, degenerative muscle disorder caused by mutations in the acid alpha-glucosidase gene, which leads to the abnormal buildup of glycogen in tissues and subsequent organ impairment. 

MZE001 is an oral glycogen synthase (GYS1) inhibitor designed to limit the buildup of disease-causing glycogen through substrate reduction therapy. The Company is currently investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of MZE001 in healthy volunteers in a phase 1 study ( Identifier: NCT05249621).

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“We are pleased to have received this designation from the FDA for MZE001, which underscores the need for new, innovative treatments for Pompe disease,” said Jason Coloma, PhD, CEO of Maze. “MZE001 is a novel mechanism we believe could be beneficial both as a monotherapy for late-onset patients as well as complement to enzyme replacement therapies across the disease spectrum.”

The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals. 


Maze Therapeutics announces FDA Orphan Drug designation granted to MZE001 for the treatment of Pompe disease. News release. Maze Therapeutics, Inc. Accessed August 31, 2022.

This article originally appeared on MPR