Olipudase alfa (XENPOZYME®) is a recombinant human acid sphingomyelinase that has been developed by Sanofi, for the treatment of acid sphingomyelinase deficiency (ASMD). Olipudase alfa catalyses the hydrolysis of sphingomyelin accumulated in hepatocytes and in mononuclear-macrophage cells, such as the lungs, liver, spleen, kidneys and bone marrow. Olipudase alfa was approved in Japan under the SAKIGAKE designation on 28 March 2022 for use in adult and paediatric patients with non-CNS manifestations of ASMD and has received a positive Committee for Medicinal Products for Human Use opinion in the EU. Regulatory review in the USA is underway. This article summarizes the milestones in the development of olipudase alfa leading to this first approval for the treatment of patients with ASMD.
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