FDA approves Xenpozyme, first treatment for rare acid sphingomyelinase deficiency

The FDA has approved Xenpozyme as the first treatment for patients with acid sphingomyelinase deficiency with symptoms unrelated to the central nervous system.Acid sphingomyelinase deficiency (ASMD) is a rare condition caused by the lack of an enzyme necessary to break down a complex lipid — sphingomyelin — which collects in the liver, spleen, lung and brain. Patients with ASMD often have enlarged abdomens that can cause pain, vomiting, feeding difficulties and increased risk for falls, as well as accompanying abnormal liver and blood tests.Xenpozyme (olipudase alfa, Genzyme) isRead More