Xenpozyme

Olipudase alfa (Xenpozyme) is an enzyme replacement therapy, for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. Acid sphingomyelinase deficiency (ASMD) is an autosomal recessive lysosomal storage disorder caused by a mutation on the sphingomyelin phophodiesterase-1 (SMPD1) gene. It is characterized by accumulation of undegraded sphingomyelin in the spleen, liver, lungs, bone marrow, and brain due to a deficiency or insufficient activity of the enzyme ASM. Symptoms may include lack of muscle coordination, brain degeneration, learning problems, loss of muscle tone, increased sensitivity to touch, spasticity, feeding and swallowing difficulties, slurred speech, and an enlarged liver and spleen. ASMD is also known as Niemann-Pick disease (NPD) types A and B. NPD type C is now considered a separate disorder, distinct from NPD types A and B. <a id="