|Other names||GZ402665, olipudase alfa-rpcp|
|Drug class||Enzyme replacement|
|Main uses||Acid sphingomyelinase deficiency (ASMD)|
|Side effects||Headache, cough, diarrhea, low blood pressure, itchiness|
|Chemical and physical data|
|Molar mass||63632.01 g·mol−1|
Olipudase alfa, sold under the brand name Xenpozyme, is a medication used to treat of acid sphingomyelinase deficiency (ASMD), previously known as Niemann-Pick disease type A/B or B. Specifically it is used for non-central nervous system (CNS) symptoms. It is given by gradual injection into a vein.
Common side events include headache, cough, diarrhea, low blood pressure, and itchiness. Other side effects may include infusion reactions, liver problems, and anaphylaxis. Use in pregnancy may harm the baby. It is an enzyme replacement, being manufactured acid sphingomyelinase (ASM), and thereby improves metabolism of fats.
Olipudase alfa was approved for medical use in Japan, Europe, and the United States in 2022. It is also approved in the United Kingdom. In the United States it costs about 7,500 USD per 20 mg as of 2022. It is made by recombinant DNA technology.
The highest potential for benefit is in people with chronic visceral forms of ASMD (type B).
Historically referred to as Niemann-Pick disease types A (NPD A) and B (NPD B), ASMD is a genetic disorder. It belongs to the larger family of metabolic disorders called lysosomal storage diseases, in which fats build up within the parts of the body's cells that break down nutrients and other materials. This affects the way cells work and causes them to die, affecting normal functioning of tissues and organs. ASMD is seriously debilitating and life-threatening since the build-up of fatty substances can cause brain damage and swelling of organs such as liver and spleen.
Xenpozyme was the first ASMD-specific treatment.
Society and culture
Olipudase alfa was approved for medical use in Japan in March 2022.
On 19 May 2022, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Xenpozyme, intended for the treatment of non-central nervous system (CNS) manifestations of acid sphingomyelinase deficiency (ASMD) type A/B or type B. Xenpozyme was reviewed under the accelerated assessment program of the European Medicines Agency (EMA). The applicant for this medicinal product is Genzyme Europe BV. Olipudase alfa was approved for medical use in the European Union in June 2022.
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- Diaz GA, Jones SA, Scarpa M, Mengel KE, Giugliani R, Guffon N, et al. (August 2021). "One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency". Genet Med. 23 (8): 1543•1550. doi:10.1038/s41436-021-01156-3. PMC 8354848. PMID 33875845.
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- Wasserstein MP, Diaz GA, Lachmann RH, Jouvin MH, Nandy I, Ji AJ, et al. (September 2018). "Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months". J Inherit Metab Dis. 41 (5): 829•838. doi:10.1007/s10545-017-0123-6. PMC 6133173. PMID 29305734.