FDA has approved alglucosidase alfa for children aged
8 years and older who have late-onset Pompe’s disease, a rare genetic
disorder that causes heart and skeletal muscle weakness, according to an FDA
The disease occurs in an estimated one in every 40,000 to 300,000 births
and eventually progresses to respiratory weakness and death due to respiratory
Alglucosidase alfa (Lumizyme, Genzyme) works by replacing acid
alpha-glucosidase, an enzyme necessary to help the heart and muscle cells to
convert glycogen into energy that patients with Pompe’s disease lack.
Without this enzyme action, researchers believe glycogen builds up in the cells
and weakens the heart and muscles.
Alglucosidase alfa has received approval with the FDA’s risk
evaluation and mitigation strategy and will only be available through an
alglucosidase alfa control and education program (Lumizyme ACE), a distribution
system that will ensure that the medication reaches the correct patient group.
The only other Pompe’s disease treatment currently approved in the
United States is Myozyme (Genzyme), which has been in short supply due to
limited manufacturing capacity, according to the press release, and has been
reserved for younger patients with more aggressive forms of the disease.
“Pompe’s disease is a devastating condition without the
appropriate treatment,” Julie Beitz, MD, director of the Office of
Drug Evaluation III in the
FDA’s Center for Drug Evaluation and Research,
said in the press release. “The approval of Lumizyme will provide an
important treatment for patients diagnosed later in life with Pompe’s
Alglucosidase alfa will carry a Boxed Warning due to risk for
anaphylaxis, severe allergic reactions and
immune-mediated reactions, according to the press release. The most commonly
reported adverse effects were infusion-related reactions, hives, diarrhea,
vomiting, shortness of breath, itchy skin, rash, neck pain, partial hearing
loss, flushing, pain in extremities and chest discomfort.