New agent approved for late-onset Pompe disease

FDA has approved alglucosidase alfa (Lumizyme, Genzyme) for patients aged 8 years and older for the treatment of late-onset (noninfantile) Pompe disease.

The agent was approved with a risk evaluation and mitigation strategy and will be available only through a restricted distribution system. The product labeling will also contain a boxed warning regarding an increased risk of anaphylaxis and severe allergic and immune-mediated reactions.

The approval of this agent was based on a clinical study in 90 patients aged 10 to 70 years with late-onset Pompe disease. In this study, the most common adverse events included infusion-related reactions.