First therapy to treat two types of Niemann-Pick disease

"EMA has recommended granting a marketing authorisation in the European Union (EU) for Xenpozyme (olipudase alfa), a therapy for the treatment of non-central nervous system (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD), a rare and progressive genetic disease. Xenpozyme is indicated for use in children and adults of all ages with type A/B or type B ASMD. " Read more on the EMA website:

First therapy to treat two types of Niemann-Pick disease, a rare genetic metabolic disorder | European Medicines Agency (europa.eu)