Sanofi’s Rare Disease Drug Xenpozyme Wins World’s First Approval in Japan
Sanofi’s Xenpozyme, also known as olipudase alfa, the world's first therapy for the rare disease Niemann-Pick type A/B and B, has been approved for the treatment of children and adults with non-CNS manifestations of Acid Sphingomyelinase Deficiency (ASMD) by Japan's drug regulator. Sanofi hopes to receive approvals for the drug in the United States and Europe before the end of the year. Xenpozyme is a recombinant human Acid Sphingomyelinase (ASM) enzyme designed to replace deficient or defective ASM. It is only approved in Japan for the treatment of ASMD types A/B and B, and it has not been s...