Muscling up on Myozyme

Oxyrane U.K. Ltd. and BioMarin Pharmaceutical Inc. have separately reported on their next-generation enzyme replacement therapies for Pompe's disease. Both molecules cleared glycogen from mouse muscle better than the marketed drug Myozyme from Sanofi.^1,2 BioMarin has moved its therapy into a Phase I/II trial, whereas Oxyrane is carrying out IND-enabling animal toxicology studies.

Pompe's disease is an inherited disorder of glycogen metabolism caused by an absence of or deficiency in the lysosomal enzyme acid α-glucosidase (GAA). The resulting accumulation of glycogen in cardiac and skeletal muscle leads to severe and progressive muscle weakness, cardiomyopathy and respiratory failure...