The European regulator approved Nexviadyme to treat the full spectrum of Pompe disease and Xenpozyme to treat non-Central Nervous System (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD)


Flags in front of the European Commission building in Brussels. (Credit: Sébastien Bertrand/Wikipedia)

French healthcare company Sanofi has received separate regulatory approvals from the European Commission (EC) for two of its enzyme replacement therapies to treat rare diseases.

The European regulator approved Nexviadyme (avalglucosidase alfa) to treat the full spectrum of both infantile-onset Pompe disease (IOPD) and late-onset Pompe disease (LOPD).

Pompe disease is a rare, progressive and severe muscle disorder, characterised by low levels of the enzyme acid alpha-glucosidase (GAA) resulting in the build-up of glycogen, leading to irreversible damage to skeletal and cardiac muscles.

Sanofi said that Nexviadyme is specifically designed to target the mannose-6-phosphate (M6P) receptor to help improve uptake and enhance glycogen clearance in target tissues.

The enzyme replacement therapy (ERT) has been approved to treat Pompe disease in the EU, the US, Japan, Canada, Switzerland, Australia, Brazil, Taiwan and the UAE.

Sanofi speciality 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 recognised and made available to patients in Europe and beyond.”

Separately, the EC has also approved Xenpozyme (olipudase alfa) to treat non-Central Nervous System (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD) in adults and children with ASMD type A/B or ASMD type B.

ASMD is a rare, progressive genetic disease characterised by an enlarged spleen or liver, difficulty breathing, lung infections, and unusual bruising or bleeding, among other disease manifestations.

Xenpozyme is an ERT specifically designed to replace the deficient acid sphingomyelinase (ASM), an enzyme that facilitates the breakdown of the lipid sphingomyelin.

The EC approval is based on positive results from the ASCEND and ASCEND-Peds clinical trials, in which Xenpozyme showed superior outcomes in lung function.

In the clinical trials, treatment using Xenpozyme improvement in lung function, along with reduction of spleen and liver volumes, with a well-tolerated safety profile.

The drug has received PRIME designation from the European Medicines Agency (EMA), along with breakthrough designations from other regulatory agencies worldwide, said the French firm.

Sanofi research and development global head and executive vice president John Reed said: “The ASMD community has waited many years for a treatment for this rare and debilitating genetic disease.

“The approval of Xenpozyme by the European Commission represents a transformational shift in what we can offer to patients, demonstrated by the clinically important improvements across major manifestations of ASMD and the sustained effects noted over longer-term treatment.”