The Japanese Ministry of Health, Labor, and Welfare (MHLW) approved Sanofi's (NASDAQ:SNY) Xenpozyme (olipudase alfa) to treat adult and pediatric patients with non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD).
ASMD is a rare, progressive, and potentially life-threatening genetic disease caused due to deficiency of the enzyme acid sphingomyelinase, which is required to break down a fatty substance (lipid) called sphingomyelin.
The company said Xenpozyme is currently the only approved treatment for ASMD and was approved under Japan's regulatory fast-track pathway SAKIGAKE.
The approval was backed by positive data from the ASCEND and ASCEND-Peds clinical trials.
The company said that Xenpozyme is currently under priority review in the U.S. — with decision expected in early Q3 — and in the EU, with a decision expected in H2.