The Japan Ministry of Health, Labor and Welfare has authorized Xenpozyme for the treatment of adult and pediatric patients with non-central nervous system manifestations of acid sphingomyelinase deficiency.
Xenpozyme (olipudase alfa, Sanofi), a recombinant human acid sphingomyelinase enzyme, is currently the first and only approved treatment for acid sphingomyelinase deficiency (ASMD), a rare, progressive and potentially life-threatening genetic disease, the company said in a press release.
“The approval of Xenpozyme is a watershed moment for ASMD patients and their families, representing