- Lumizyme was originally approved in 2010 was for the treatment of Pompe disease in children aged eight and older.
- Pompe disease is a rare genetic disorder that manifests as heart and skeletal weaknesses and progresses into respiratory failure. It affects every 40,000 to 300,000 births.
- The FDA expanded the label for Lumizyme in response to new data that showing the drug is also safe and effective in children under age eight.
REMS (risk evaluation and mitigation strategy) programs are structured in many different ways. The REMS program for Lumizyme, known as the Lumizyme ACE Program, communicated the product’s risks, including anaphylaxis, severe allergic reactions and severe skin and immune-mediated reactions. This messaging was aimed at both physicians and patients. While children eight and older with Pompe disease -- most of whom had what was considered late-onset disease -- were treated with Lumizyme, children under eight, including those with infantile-onset disease, were treated with Myozyme, which is also manufactured by Genzyme.
After extensive testing, it is now clear that Myozyme and Lumizyme are chemically and biochemically comparable. They are produced from the same cell lines, but at different production scales. Furthermore, results of a study of 18 infantile-onset patients treated with Lumizyme confirmed the treatment's efficacy and safety in this population. Despite the fact that some restrictions have been removed, a boxed warning will still be part of Lumizyme’s label.