European Commission approves potential new treatment for Pompe disease

The European Commission (EC) has issued a marketing authorisation for Sanofi Genzyme’s therapy Nexviadyme (avalglucosidase alfa) for the long-term treatment of both late-onset and infantile-onset Pompe disease.

Pompe disease is a rare, progressive and debilitating muscle disorder which can present as infantile-onset Pompe disease (IOPD), the most severe form of the disease with rapid onset in infancy, or as late-onset Pompe disease (LOPD), which results in progressive muscle damage.