Xenpozyme Approved by EC for the Treatment of ASMD

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The European Commission has approved Xenpozyme as the first treatment for ASMD.

Sanofi announced on June 28, 2022 that the European Commission (EC) has approved Xenpozyme (olipudase alfa) as the first enzyme replacement therapy for the treatment of Acid Sphingomyelinase Deficiency (ADMS). The European Medicines Agency (EMA) granted the treatment PRIME designation due to the urgent unmet medical needs of the ASMD community.

ASMD is a rare, progressive genetic disease with significant morbidity and mortality, especially among infants and children. Many pediatric patients with the disease do not survive to adulthood. ASMD symptoms may manifest as enlarged spleen or liver, difficulty breathing, lung infections, and unusual bruising or bleeding. Supportive care is the only current option to aid in managing the symptoms of the disease.

Xenpozyme is an enzyme replacement therapy that is designed to replace deficient or defective acid sphingomyelinase (ASM), an enzyme that allows for the breakdown of lipid sphingomyelin. ASMD can lead to lifelong damage to multiple organs due to the insufficient amount of the ASM enzyme.


“The ASMD community has waited many years for a treatment for this rare and debilitating genetic disease,” said John Reed, MD, PhD, executive vice president and global head of research and development at Sanofi, in a press release. “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.”

Source: Sanofi