Genzyme's Lumizyme Now Indicated for Infantile-Onset Pompe Disease
FDA will no longer require Genzyme to continue the Risk Evaluation and Mitigation Strategy program.
FDA will no longer require Genzyme to continue the Risk Evaluation and Mitigation Strategy program.
On Aug. 1, the U.S. Food and Drug Administration (FDA)
Furthermore, THE FDA will no longer require Genzyme to continue the Risk Evaluation and Mitigation Strategy (REMS) known as the Lumizyme ACE (Alglucosidase Alfa Control and Education) Program.
Pompe disease is a lysosomal storage disease characterized by progressive skeletal muscle weakness and respiratory insufficiency. It is caused by a deficiency in GAA activity, which leads to accumulation of glycogen in tissues affected by the disease (primarily heart and muscle). Persons with late-onset Pompe tend to have a milder form of the condition while those with infantile-onset Pompe usually have more severe symptoms and die at a fairly young age if not treated.
Lumizyme is produced on a large scale (4000L) and until today, indicated only for treating patients with late-onset Pompe disease older than 8 years of age in the United States. Children with infantile-onset Pompe disease were given
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