Xenpozyme

XENPOZYME® (olipudase alfa-rpcp) is an enzyme replacement therapy (ERT) for the treatment of non-CNS manifestations of ASMD in both adults and pediatric patients. Mechanism: Recombinant human acid sphingomyelinase that hydrolyzes accumulated sphingomyelin in lysosomes, reducing lipid storage. Disease background: ASMD (Niemann-Pick disease types A/B) is an autosomal recessive lysosomal storage disorder caused by SMPD1 gene mutations, leading to deficient ASM activity, sphingomyelin buildup, hepatosplenomegaly, pulmonary dysfunction, and systemic morbidity. Clinical trials (ASCEND, ASCEND-Peds): demonstrated significant reductions in spleen/liver volume and improvements in pulmonary function (DLCO). FDA Indication: Approved for non-CNS manifestations of ASMD (July 2022; PI revised December 2023).