The US Food and Drug Administration has approved Lumizyme (alglucosidase alfa) to treat patients aged eight years and older with Pompe disease.
The genetic disorder is characterised by heart, skeletal and respiration weakness, often leading to death from respiratory failure.
Lumizyme works by replacing deficient acid alpha-glucosidase, an enzyme necessary for proper muscle GAA that is prevented by the gene mutation that characterises Pompe disease, thereby reducing the accumulated glycogen in heart and skeletal muscle cells.
Lumizyme has been approved with a risk evaluation and mitigation strategy and is available through a restricted distribution system to ensure that the drug is accessible only to the relevant patient group.
The safety and efficacy of Lumizyme was evaluated in a clinical study with 90 patients, ranging from ten years to 70 years.