Sanofi has received marketing authorisation from the European Commission (EC) for Nexviadyme (avalglucosidase alfa) to treat the complete spectrum of infantile-onset Pompe disease (IOPD) and late-onset Pompe disease (LOPD).
The regulator has approved the therapy as a potential new standard of care to treat Pompe disease, a rare, progressive and severe muscle disorder.
Individuals with this disease have low levels of the enzyme acid alpha-glucosidase (GAA) that results in glycogen build-up. This causes irreversible damage to skeletal and cardiac muscles.
Nexviadyme is an enzyme replacement therapy (ERT) which has been specifically designed to target the mannose-6-phosphate (M6P) receptor.
M6P receptor is an important pathway for cellular uptake of ERT and transport to the lysosome.
The therapy helps to improve uptake and enhance glycogen clearance in target tissues.
It has been approved to treat Pompe disease in Canada, Taiwan, Switzerland, Japan, Australia, Brazil, the US, the UAE, and the EU.
Sanofi Specialty Care executive vice-president Bill Sibold said: “For more than two decades, we’ve been working with the community and leveraging our scientific expertise to improve care for people living with Pompe disease.
“We strongly believe in the meaningful clinical benefits of this medicine as a new standard of care and will work hard to ensure the broadest possible access in Europe despite the European Commission’s failure to recognize Nexviadyme’s NAS and OMP designations.
“We call on patient advocacy groups, policymakers, clinicians and patients to join us in our efforts to ensure innovative treatments are appropriately recognized and made available to patients in Europe and beyond.”