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:
The Warnings and Precautions section of the Lumizyme product label and the Clinical Studies section of the Lumizyme label have been updated to include the safety information of the drug in infantile-onset Pompe disease patients. This includes information from the currently approved Myozyme label and information from a new, uncontrolled study in which patients with infantile onset disease were treated with Lumizyme.
Lumizyme is approved with a Boxed Warning because of the risk of anaphylaxis, severe allergic reactions, immune-mediated reactions and cardiorespiratory failure.
Health care professionals should continue to refer to the drug prescribing information for the latest recommendations on prescribing Lumizyme and report adverse events to the FDA’s MedWatch program (http://www.fda.gov/Safety/MedWatch/default.htm).
Distribution of Lumizyme will no longer be restricted. Health care professionals, healthcare facilities, and patients will no longer be required to enroll in the Lumizyme REMS program (Lumizyme ACE Program) to be able to prescribe, dispense, or receive Lumizyme.