FAQs about enzyme replacement therapy for Pompe disease

While other treatments may be administered to help manage the symptoms of Pompe disease, enzyme replacement therapies and the combination approach Pombiliti + Opfolda — which contains a version of the GAA enzyme alongside an enzyme stabilizer — are the only approved treatments that directly address the root cause of the disorder in order to slow disease progression.

There are two enzyme replacement therapies approved in the U.S.: Lumizyme and Nexviazyme. Lumizyme is approved for all forms of Pompe disease, while Nexviazyme is approved for patients with late-onset disease, ages 1 and older. Another approved therapy, Pombiliti + Opfolda, is a combination of a lab-made form of the GAA enzyme alongside an enzyme stabilizer. It is approved for certain adults with late-onset Pompe disease who are not improving on their current ERT.

Both approved enzyme replacement therapies, Lumizyme and Nexviazyme, are administered intravenously, or via an infusion into the bloodstream, every other week. Pombiliti, the lab-made form of the GAA enzyme given in a combination treatment with the enzyme stabilizer Opfolda, is also given every two weeks (Opfolda is taken about an hour before each Pombiliti infusion).

Enzyme replacement therapy is not a cure for Pompe disease, but it can slow the disease’s progression.