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12:00 AM
Dec 12, 2011
 |  BioCentury  |  Product Development

Muscling in on Myozyme

Oxyrane aims to compete with Genzyme's Myozyme in Pompe's

Oxyrane U.K. Ltd. is developing a next-generation enzyme replacement therapy for Pompe's disease that could improve on Myozyme. By enhancing delivery of enzyme to diseased tissue, the company hopes to reduce dosage and infusion times and lower cost to patients and payers.

Pompe's is an inherited disorder of glycogen metabolism caused by an absence of or deficiency in the lysosomal enzyme acid alpha glucosidase (GAA). The resulting accumulation of glycogen in tissues, especially cardiac and skeletal muscle, leads to severe and progressive muscle weakness, cardiomyopathy and respiratory failure. The disease has an estimated incidence of 1 in 40,000.

Sanofi's Genzyme Corp. unit markets two drugs to treat Pompe's, Myozyme and Lumizyme. Both are recombinant forms of human GAA that are manufactured in CHO cell lines.

The drugs must be given at high concentrations to ensure sufficient enzyme enters skeletal muscle cells, which are much less accessible to systemic enzyme replacement therapy than the cells targeted in other lysosomal storage diseases, such as macrophages and endothelial cells.

The recommended dose of the Genzyme drugs is 20 mg/kg biweekly delivered as an intravenous infusion over four hours.

However, the dose must...

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