FDA approves Lumizyme to treat infantile-onset Pompe disease

Alglucosidase alfa was today deemed safe by the FDA for treating patients younger than 8 years old who have Pompe disease and its Risk Evaluation and Mitigation Strategy is now slated for elimination, according to a press release.

Approved in 2010 to treat patients with late-onset Pompe disease, the lysosomal glycogen-specific enzyme, alglucosidase alfa (Lumizyme, Genzyme), was approved for infantile-onset following new safety and efficacy evidence.

Children younger than age 8 years with the rare genetic disorder, which causes mutations that prevent the body from making enough functional acid alpha-glucosidase (GAA), have until now been treated with Myozyme, another alglucosidase alfa manufactured by Genzyme. The drugs are produced from the same cell line at different production scales.

Lumizyme infusions are believed to replace deficient GAA and reduce glycogen that has accumulated in heart and skeletal muscle cells.

Newly available information shows Lumizyme and Myozyme are chemically and biochemically comparable and, therefore, expected to be equally safe and effective, according to the release. A trial of 18 patients with infantile-onset, aged between 0.2 and 5.8 months at initial therapy, provides additional support that children will have similar improvements as they have experienced to date.

The data submitted makes the Risk Evaluation and Mitigation Strategy unnecessary; it was initially required to diminish rapid-disease progression in the youngest children and communicate the risks of anaphylaxis, severe allergic reactions and skin and immune reactions. The risks will continue to be communicated through packaging, according to the release.

Commonly reported side effects of Lumizyme have been infusion-related reactions, including severe allergic reactions, hives, diarrhea, vomiting, shortness of breath, itchy skin, skin rash, neck pain, partial hearing loss, flushing, pain in extremities and chest discomfort.

For more information: FDA expands approval of drug to treat Pompe disease to patients of all ages; removes risk mitigation strategy requirements