Pompe Disease Drug Now Approved for All Ages

The FDA announced the approval of Lumizyme (alglucosidase alfa; Genzyme) for the treatment of patients with infantile-onset Pompe disease, including patients who are <8 years old. Along with the approval, the Risk Evaluation and Mitigation Strategy (REMS) is being removed.

Lumizyme was approved in 2010 with a REMS to limit its use to treatment of pateints who were >8 years old. The REMS communicated the risks of anaphylaxis, severe allergic reactions, and severe skin and systemic immune mediated reactions to prescribers and patients.

The FDA reviewed newly available information and concluded that Lumizyme and Myozyme are chemically and biochemically comparable. Further, the safety and efficacy of Lumizyme and Myozyme (alglucosidase alfa) are also expected to be comparable. A separate single-center clinical study supported that patients with infantile-onset Pompe disease treated with Lumizyme will have a similar improvement in ventilator-free survival as those treated with Myozyme.

Lumizyme is a recombinant human alpha-glucosidase enzyme that is thought to work by replacing the deficient alpha-glucosidase (GAA), thereby reducing the accumulated glycogen in heart and skeletal muscle cells.

Want to read more?