http://www.raredr.com/news/genzyme-lumizyme-for-infantile-onset-pompe
Genzyme's Lumizyme Now Indicated for Infantile-Onset Pompe Disease

RDR Staff


Today, the U.S. Food and Drug Administration (FDA) announced the expansion of Genzyme’s Lumizyme (alglucosidase alfa) to include patients with infantile-onset Pompe disease, including patients  less than 8 years of age. Until today, Lumizyme was only indicated for persons with late-onset Pompe disease.

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 Myozyme, which is the same drug as Lumizyme but produced on a smaller, and more costly, scale (160L).  The expansion of the label for Lumizyme will likely allow for more efficient production and distribution of the drug as the FDA concluded that the manufacturing of both drugs are comparable and the safety profiles similar.

In a press release, Genzyme President and CEO, David Meeker, M.D. “We are pleased we can now offer alglucosidase alfa produced at the 4000L scale to all patients in the US,” adding, “We are thankful to the entire Pompe community who has been on this journey to provide a sustainable, long-term option for Pompe patients in the United States.”

The FDA also noted:
 
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