Approved Treatments for Pompe Disease

Enzyme replacement therapy, commonly abbreviated as ERT, is an approved treatment strategy used in Pompe disease. ERTs are currently the only approved treatments for Pompe that address the condition’s underlying cause. By replacing needed enzymes, the therapy works to reduce and slow the progression of muscle wasting and heart problems that characterize Pompe disease.

Lumizyme (alglucosidase alfa) is an enzyme replacement therapy approved for Pompe disease. It works to ease breathing and improve motor function in patients with the genetic disorder.

Nexviazyme (avalglucosidase alfa) is an approved treatment for Pompe disease, developed by Sanofi, which has been shown to ease breathing and improve walking ability in people with the genetic disorder. It is marketed under the name Nexviadyme in Europe.

Pombiliti + Opfolda (cipaglucosidase alfa/miglustat), formerly known as AT-GAA, is an approved combination therapy for adults with late-onset Pompe disease, or LOPD. The treatment works mainly to enhance walking ability and improve or stabilize respiratory function.