| Clinical data | |
|---|---|
| Trade names | Xenpozyme |
| Other names | GZ402665, olipudase alfa-rpcp |
| License data | |
| Pregnancy category |
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| Routes of administration | Intravenous |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| UNII | |
| KEGG | |
| Chemical and physical data | |
| Formula | C2900H4373N783O791S24 |
| Molar mass | 63632.01 g·mol−1 |
Olipudase alfa, sold under the brand name Xenpozyme, is a medication used for the treatment of non-central nervous system (CNS) manifestations of acid sphingomyelinase deficiency (ASMD) type A/B or type B.[2][4][3][5]
The common side events include infections, infusion-related reactions, or gastrointestinal complaints (disease signs and symptoms in children).[5]
Historically referred to as Niemann-Pick disease types A (NPD A) and B (NPD B), ASMD is a genetic disorder.[3] 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.[3] This affects the way cells work and causes them to die, affecting normal functioning of tissues and organs.[3] 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.[3]
Xenpozyme is the first ASMD-specific treatment.[3] This medicine is an enzyme replacement therapy, developed to replace patients' deficient or defective enzyme, acid sphingomyelinase (ASM), and thereby reduce fat accumulation within cells and relieve some of the symptoms of the disease.[3] Xenpozyme was approved by the European Medicines Agency for the treatment of Acid sphingomyelinase deficiency (ASMD) type A/B or type B,[6] and by the US Food and Drug Administration for the treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients.[7] The replacement enzyme is produced by a method known as recombinant DNA technology: it is made by cells into which a gene (DNA) has been introduced, that enables them to produce the enzyme.[3]
Olipudase alfa was approved for medical use in Japan in March 2022,[4] in the European Union in June 2022,[3] and in the United States in August 2022.[2][8] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[9][10]
Society and culture[edit]
Olipudase alfa is the international nonproprietary name (INN).[11]
Legal status[edit]
Olipudase alfa was approved for medical use in Japan in March 2022.[4]
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.[3] Xenpozyme was reviewed under the accelerated assessment program of the European Medicines Agency (EMA).[3] The applicant for this medicinal product is Genzyme Europe BV.[3] Olipudase alfa was approved for medical use in the European Union in June 2022.[3][12]
References[edit]
- ^ a b "Xenpozyme APMDS". Therapeutic Goods Administration (TGA). 6 September 2023. Archived from the original on 2 January 2024. Retrieved 7 March 2024.
- ^ a b c "Xenpozyme- olipudase alfa-rpcp injection, powder, lyophilized, for solution". DailyMed. 31 August 2022. Archived from the original on 16 October 2022. Retrieved 16 October 2022.
- ^ a b c d e f g h i j k l m n "Xenpozyme EPAR". European Medicines Agency. 13 April 2022. Archived from the original on 30 July 2022. Retrieved 30 July 2022. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ^ a b c "Xenpozyme (olipudase alfa) approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency" (Press release). Sanofi. 28 March 2022. Archived from the original on 21 May 2022. Retrieved 20 May 2022.
- ^ a b "First therapy to treat two types of Niemann-Pick disease, a rare genetic metabolic disorder". European Medicines Agency (EMA) (Press release). 20 May 2022. Archived from the original on 20 May 2022. Retrieved 20 May 2022. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ^ "www.ema.europa.eu". Archived from the original on 30 July 2022. Retrieved 30 July 2022.
- ^ "www.accessdata.fda.gov" (PDF). Archived (PDF) from the original on 8 January 2023. Retrieved 24 August 2023.
- ^ "Xenpozyme: FDA-Approved Drugs". U.S. Food and Drug Administration (FDA). Archived from the original on 3 September 2022. Retrieved 2 September 2022.
- ^ "Advancing Health Through Innovation: New Drug Therapy Approvals 2022". U.S. Food and Drug Administration (FDA). 10 January 2023. Archived from the original on 21 January 2023. Retrieved 22 January 2023.
This article incorporates text from this source, which is in the public domain.
- ^ New Drug Therapy Approvals 2022 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2024. Archived from the original on 14 January 2024. Retrieved 14 January 2024. This article incorporates text from this source, which is in the public domain.
- ^ World Health Organization (2015). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 73". WHO Drug Information. 29 (1). hdl:10665/331088.
- ^ "Xenpozyme Product information". Union Register of medicinal products. Archived from the original on 4 March 2023. Retrieved 3 March 2023.
Further reading[edit]
- 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.
- Maines E, Franceschi R, Rizzardi C, Deodato F, Piccoli G, Gragnaniello V, et al. (2022). "Atherogenic lipid profile in patients with Niemann-Pick disease type B: What treatment strategies?". J Clin Lipidol. 16 (2): 143–154. doi:10.1016/j.jacl.2022.01.008. PMID 35181260. S2CID 246974107.
- 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.
