The Food and Drug Administration has just approved a new drug to treat those with late-onset Pompe disease. It's called alglucosidase alfa (Lumizyme).
Pompe disease affects between 5,000 and 10,000 people globally. It's a rare lysosomal storage disorder caused by a lack of an enzyme called acid alpha-1,4-glucosidase. This lack leads to a build-up of glycogen, which eventually causes muscle damage, and can be fatal when the respiratory and cardiovascular systems are affected.
Lumizyme replaces the necessary alpha-glucosidase (GAA) in the body, reducing the glycogen buildup. The drug, which was approved based on a trial of 90 patients between 10 and 70 years old, has not been approved for younger patients with Pompe disease. The other effective drug on the market, Myozyme -- which has been approved for younger patients -- is currently in short supply and is being reserved for those with Pompe disease under 8 years old.