FDA expands approval for Lumizyme

SILVER SPRING, Md. — The Food and Drug Administration approved Lumizyme (alglucosidase alfa) for treatment of patients with infantile-onset Pompe disease, including patients who are younger than 8 years of age, the agency said. Additionally, the FDA announced that the risk evaluation and mitigation strategy that is referred to as the Lumizyme ACE program is being discontinued.



Pompe disease is a rare genetic disorder that occurs in an estimated 1-in-every-40,000 to -300,000 births. Heart and skeletal muscle weakness is the primary symptom, which progresses to respiratory weakness and death from respiratory failure.



When Lumizyme was approved in 2010, there wasn't sufficient data to support the drug's safety and efficacy in the infantile-onset Pompe population, so the drug was only approved for use in late onset Pompe disease patients who are at least 8 years of age. The FDA's expanded approval provides access to the drug for all patients with Pompe disease, regardless of age. Lumizyme is manufactured by Genzyme Corp.