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Label expansion for Lumizyme in the United States for the treatment of Pompe disease


The Food and Drug Administration ( FDA ) has approved a supplement to expand the indication for Lumizyme ( Alglucosidase alfa ). Lumizyme manufactured at the 4000L scale is now indicated for all Pompe patients of any age or phenotype.
The approval of this indication is now consistent with that of the rest of the world, where Alglucosidase alfa manufactured at the 4000L is the only scale available.
Previously, in the United States, Lumizyme had been approved only for patients with late onset Pompe disease.

In the United States, Alglucosidase alfa is manufactured at two different production scales. Alglucosidase alfa manufactured at the 160L scale ( initial pilot scale ) has a brand name of Myozyme ( Alglucosidase alfa ) and Alglucosidase alfa manufactured at the 4000L ( final manufacturing scale ) has a brand name of Lumizyme.

Based on the biochemical and clinical data provided as part of the submission, FDA concluded that Alglucosidase alfa manufactured at both scales in the US ( i.e., Lumizyme and Myozyme ) are comparable. The overall safety profile of Alglucosidase alfa remains unchanged.

Specific updates to the Lumizyme product label include: a) Updated Indication: Lumizyme ( Alglucosidase alfa ) is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease ( GAA deficiency ); b) Inclusion of safety and efficacy data from infantile-onset studies; c) Removal of the REMS program; d) Update to the boxed warning to include infantile-onset specific warning regarding fluid overload; e) Updated to Pregnancy Category C classification.

Lumizyme, marketed as Myozyme outside of U.S., is approved in more than 65 countries.

Pompe disease is a progressive, debilitating and often fatal neuromuscular disease caused by a genetic deficiency or dysfunction of the lysosomal enzyme acid alpha-glucosidase ( GAA ) affecting an estimated 1 in 40,000 people worldwide. This enzymatic defect results in the accumulation of glycogen primarily in muscle tissues that leads to muscle weakness, loss of respiratory function, and often premature death. Absent treatment, when symptoms occur in infancy, babies typically die within the first year of life. When symptoms occur in childhood or adulthood, patients often lose their ability to walk and require wheelchairs to assist with mobility and experience difficulty breathing as well as mechanical ventilation to breathe. ( Xagena )

Source: Genzyme, 2014

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