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Olipudase Alfa: First Approval.
Drugs. 2022 Jun; 82(8):941-947.D

Abstract

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.

Links

Publisher Full Text (DOI)

Authors+Show Affiliations

Keam SJ
Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.

MeSH

AdultChildDrug ApprovalEnzyme Replacement TherapyHumansNiemann-Pick Disease, Type ARecombinant ProteinsSphingomyelin Phosphodiesterase

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

35639287

Citation

Keam, Susan J.. "Olipudase Alfa: First Approval." Drugs, vol. 82, no. 8, 2022, pp. 941-947.
Keam SJ. Olipudase Alfa: First Approval. Drugs. 2022;82(8):941-947.
Keam, S. J. (2022). Olipudase Alfa: First Approval. Drugs, 82(8), 941-947. https://doi.org/10.1007/s40265-022-01727-x
Keam SJ. Olipudase Alfa: First Approval. Drugs. 2022;82(8):941-947. PubMed PMID: 35639287.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Olipudase Alfa: First Approval. A1 - Keam,Susan J, PY - 2022/6/1/pubmed PY - 2022/6/23/medline PY - 2022/5/31/entrez SP - 941 EP - 947 JF - Drugs JO - Drugs VL - 82 IS - 8 N2 - 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. SN - 1179-1950 UR - https://www.unboundmedicine.com/medline/citation/35639287/Olipudase_Alfa:_First_Approval_ DB - PRIME DP - Unbound Medicine ER -