CHMP recommends Nexviadyme, a treatment for for glycogen storage disease type II

Nexviadyme will be available as a 100 mg powder for concentrate for solution for infusion. The active substance of Nexviadyme is avalglucosidase alfa, a recombinant human acid alfa-glucosidase (ATC code: not yet assigned), which is an enzyme replacement therapy that provides an exogenous source of acid alfa-glucosidase. The benefit of Nexviadyme is its ability to improve the respiratory function (also called force vital capacity) of Pompe disease patients. The most common side effects are hypersensitivity (including anaphylaxis), infusion associated reactions (pruritus, rash, headache, urticaria, fatigue, nausea and chills).The full indication is: Nexviadyme (avalglucosidase alfa) is indicated for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid alfa-glucosidase deficiency).