The U.S. Food and Drug Administration approved Xenpozyme (Olipudase alfa) for intravenous infusion in pediatric and adult patients with Acid Sphingomyelinase Deficiency (ASMD), a rare genetic disease that causes premature death. Xenpozyme is the first approved medication to treat symptoms that are not related to the central nervous system in patients with ASMD.
Xenpozyme is an enzyme replacement therapy that helps reduce sphingomyelin accumulation in the liver, spleen, and lung. The efficacy of Xenpozyme for the treatment of ASMD was demonstrated in a randomized, double-blind, placebo-controlled study of 31 patients randomized to take Xenpozyme or placebo. Because the study had the placebo comparator and measured treatment benefits that could be detected during the study’s duration, the FDA was able to conclude that Xenpozyme is effective. Overall, treatment with Xenpozyme improved lung function and reduced liver and spleen size.
