Sanofi's Xenpozyme wins European race in ASMD

The European Commission (EC) has approved Sanofi’s (Euronext: SAN) Xenpozyme (olipudase alfa) as the first and only enzyme replacement therapy to treat non-central nervous system (CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in pediatric and adult patients with ASMD type A/B or ASMD type B.

This approval is based on positive data from the ASCEND and ASCEND-Peds trials, in which Xenpozyme showed substantial and clinically-relevant improvement in lung function and reduction of spleen and liver volumes, with a well-tolerated safety profile.

"The ASMD community has waited many years for a treatment for this rare and debilitating genetic disease"ASMD is an extremely rare, progressive genetic disease with significant morbidity and mortality, especially among infants and children, as many pediatric patients will not survive to adulthood.