Xenpozyme (olipudase alfa) was the first therapy approved for acid sphingomyelinase deficiency, historically called Niemann-Pick disease types A and B, in which sphingomyelin accumulates in the liver, spleen, and lungs.
Olipudase alfa replaces the deficient enzyme, breaking down accumulated sphingomyelin to reduce organ enlargement and improve lung function. Infusions are given every two weeks with careful dose escalation, covering the non-central nervous system manifestations of acid sphingomyelinase deficiency in adults and children.
Xenpozyme holds orphan designation, remains the only approved therapy for the condition, and faces no biosimilar competition. Its inability to address the neurological form of the disease defines the remaining unmet need and the target for next-generation approaches.
