Label for Lumizyme (Alglucosidase Alfa) Expanded to Include All Patients With Pompe Disease
Previous indication was for late-onset disease
The FDA has approved a supplement to expand the indication for Lumizyme (alglucosidase alfa, Genzyme) in patients with Pompe disease.
Lumizyme (manufactured at the 4,000-L scale) is now indicated for all Pompe patients of any age or phenotype. The approval of this indication is consistent with that of the rest of the world, where alglucosidase alfa manufactured at 4,000 L is the only scale available. Previously, in the U.S., Lumizyme had been approved only for patients with late-onset Pompe disease.
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). This enzymatic defect results in the accumulation of glycogen primarily in muscle tissues, which leads to muscle weakness, loss of respiratory function, and often premature death. Without 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, often requiring mechanical ventilation to breathe. The disorder affects an estimated 1 in 40,000 people worldwide.
In the U.S., alglucosidase alfa is manufactured at two different production scales. Alglucosidase alfa manufactured at the 160-L scale (initial pilot scale) has a brand name of Myozyme (alglucosidase alfa), and alglucosidase alfa manufactured at the 4, 000-L scale (final manufacturing scale) has a brand name of Lumizyme.
Based on the biochemical and clinical data provided as part of the regulatory submission, the FDA concluded that alglucosidase alfa manufactured at both scales in the U.S. (i.e., Lumizyme and Myozyme) are comparable. The overall safety profile of alglucosidase alfa remains unchanged.
Specific updates to the Lumizyme product label include:
- Updated indication: Lumizyme (alglucosidase alfa) is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (GAA deficiency).
- Inclusion of safety and efficacy data from infantile-onset studies
- Removal of the Risk Evaluation and Mitigation Strategies (REMS) program
- Update to the boxed warning to include an infantile-onset–specific warning regarding fluid overload
- Update to Pregnancy Category C classification
The current boxed warning for Lumizyme (alglucosidase alfa) includes the potential for anaphylaxis, hypersensitivity, immune-mediated reactions, or cardiorespiratory failure.
Source: Genzyme; August 1, 2014.