Olipudase alfa explained
| Tradename: | Xenpozyme |
| Dailymedid: | Olipudase alfa |
| Pregnancy Au: | D |
| Routes Of Administration: | Intravenous |
| Atc Prefix: | A16 |
| Atc Suffix: | AB25 |
| Legal Au: | S4 |
| Legal Au Comment: | [1] |
| Legal Ca: | Rx-only |
| Legal Ca Comment: | [2] |
| Legal Us: | Rx-only |
| Legal Us Comment: | [3] |
| Legal Eu: | Rx-only |
| Legal Eu Comment: | [4] |
| Legal Status: | Rx-only |
| Cas Number: | 927883-84-9 |
| Drugbank: | DB12835 |
| Unii: | 6D5766Q4OP |
| Kegg: | D10820 |
| Synonyms: | GZ402665, olipudase alfa-rpcp |
| C: | 2900 |
| H: | 4373 |
| N: | 783 |
| O: | 791 |
| S: | 24 |
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 type A/B or type B.[5] [6]
The most common side events include infections, infusion-related reactions, or gastrointestinal complaints (disease signs and symptoms in children).
Historically referred to as Niemann-Pick disease types A (NPD A) and B (NPD B), acid sphingomyelinase deficiency 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. Acid sphingomyelinase deficiency 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 is the first acid sphingomyelinase deficiency-specific treatment. 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.
Olipudase alfa was approved for medical use in Japan in March 2022, in the European Union in June 2022, and in the United States in August 2022.[7] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[8] [9]
Medical uses
Olipudase alfa is indicated as an enzyme replacement therapy for the treatment of non-central nervous system manifestations of acid sphingomyelinase deficiency in people with type A/B or type B.
Olipudase alfa is an enzyme replacement therapy, developed to replace patients' deficient or defective enzyme, acid sphingomyelinase, and thereby reduce fat accumulation within cells and relieve some of the symptoms of the disease. Xenpozyme was approved by the European Medicines Agency for the treatment of acid sphingomyelinase deficiency type A/B or type B, and by the US FDA for the treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency.
Society and culture
Olipudase alfa is the international nonproprietary name (INN).[10]
Legal status
Olipudase alfa was approved for medical use in Japan in March 2022.
In 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 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.[11]
Further reading
- Diaz GA, Jones SA, Scarpa M, Mengel KE, Giugliani R, Guffon N, Batsu I, Fraser PA, Li J, Zhang Q, Ortemann-Renon C . 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 . August 2021 . 33875845 . 8354848 . 10.1038/s41436-021-01156-3 .
- Maines E, Franceschi R, Rizzardi C, Deodato F, Piccoli G, Gragnaniello V, Burlina A, Soffiati M . Atherogenic lipid profile in patients with Niemann-Pick disease type B: What treatment strategies? . J Clin Lipidol . 16 . 2 . 143–154 . 2022 . 35181260 . 10.1016/j.jacl.2022.01.008 . 246974107 .
- Wasserstein MP, Diaz GA, Lachmann RH, Jouvin MH, Nandy I, Ji AJ, Puga AC . 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 . September 2018 . 29305734 . 6133173 . 10.1007/s10545-017-0123-6 .
Notes and References
- Web site: Xenpozyme APMDS . Therapeutic Goods Administration (TGA) . 6 September 2023 . 7 March 2024 . 2 January 2024 . https://web.archive.org/web/20240102021342/https://www.tga.gov.au/resources/auspmd/xenpozyme . live .
- Web site: Notice: Multiple Additions to the Prescription Drug List (PDL) [2024-04-30] ]. Health Canada . 30 April 2024 . 3 May 2024.
- Web site: Xenpozyme- olipudase alfa-rpcp injection, powder, lyophilized, for solution . DailyMed . 31 August 2022 . 16 October 2022 . 16 October 2022 . https://web.archive.org/web/20221016044530/https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=01a910ee-a33e-4be3-ac41-322d64c34311 . live .
- Web site: Xenpozyme EPAR . European Medicines Agency . 13 April 2022 . 30 July 2022 . 30 July 2022 . https://web.archive.org/web/20220730022125/https://www.ema.europa.eu/en/medicines/human/EPAR/xenpozyme . live . Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- Xenpozyme (olipudase alfa) approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency . Sanofi . 20 May 2022 . 28 March 2022 . 21 May 2022 . https://web.archive.org/web/20220521063633/https://www.sanofi.com/media-room/press-releases/2022/2022-03-28%2009-00-00%202410648 . live .
- First therapy to treat two types of Niemann-Pick disease, a rare genetic metabolic disorder . European Medicines Agency (EMA) . 20 May 2022 . 20 May 2022 . 20 May 2022 . https://web.archive.org/web/20220520122046/https://www.ema.europa.eu/en/news/first-therapy-treat-two-types-niemann-pick-disease-rare-genetic-metabolic-disorder . live . Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- Web site: Xenpozyme: FDA-Approved Drugs . U.S. Food and Drug Administration (FDA) . 2 September 2022 . 3 September 2022 . https://web.archive.org/web/20220903053834/https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=761261 . live .
- Web site: Advancing Health Through Innovation: New Drug Therapy Approvals 2022 . U.S. Food and Drug Administration (FDA) . 10 January 2023 . 22 January 2023 . 21 January 2023 . https://web.archive.org/web/20230121035714/https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/new-drug-therapy-approvals-2022 . live .
- New Drug Therapy Approvals 2022 . U.S. Food and Drug Administration (FDA) . January 2024 . PDF . 14 January 2024 . https://web.archive.org/web/20240114065648/https://www.fda.gov/media/164429/download . 14 January 2024 . live .
- ((World Health Organization)) . 2015 . International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 73 . WHO Drug Information . 29 . 1 . 10665/331088 . free . World Health Organization .
- Web site: Xenpozyme Product information . Union Register of medicinal products . 3 March 2023 . 4 March 2023 . https://web.archive.org/web/20230304064738/https://ec.europa.eu/health/documents/community-register/html/h1659.htm . live .
