Olipudase alfa

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Olipudase alfa
Names
Trade namesXenpozyme
Other namesGZ402665, olipudase alfa-rpcp
Clinical data
Drug classEnzyme replacement[1]
Main usesAcid sphingomyelinase deficiency (ASMD)[2]
Side effectsHeadache, cough, diarrhea, low blood pressure, itchiness[2]
Routes of
use		Intravenous
External links
AHFS/Drugs.comMonograph
Legal
License data
Legal status
  • US: ℞-only [2]
  • EU: Rx-only [1]
  • In general: ℞ (Prescription only)
Chemical and physical data
FormulaC2900H4373N783O791S24
Molar mass63632.01 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.[2] Specifically it is used for non-central nervous system (CNS) symptoms.[2][1] It is given by gradual injection into a vein.[2]

Common side events include headache, cough, diarrhea, low blood pressure, and itchiness.[2] Other side effects may include infusion reactions, liver problems, and anaphylaxis.[2] Use in pregnancy may harm the baby.[2] It is an enzyme replacement, being manufactured acid sphingomyelinase (ASM), and thereby improves metabolism of fats.[1]

Olipudase alfa was approved for medical use in Japan, Europe, and the United States in 2022.[1][2] It is also approved in the United Kingdom.[3] In the United States it costs about 7,500 USD per 20 mg as of 2022.[4] It is made by recombinant DNA technology.[2]

Medical use

The highest potential for benefit is in people with chronic visceral forms of ASMD (type B).[1]

Historically referred to as Niemann-Pick disease types A (NPD A) and B (NPD B), ASMD is a genetic disorder.[1] 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.[1] This affects the way cells work and causes them to die, affecting normal functioning of tissues and organs.[1] 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.[1]

Dosage

It is given every two weeks.[1] In children an initial dose of 0.03 mg/kg is used while in adults it is started at 0.1 mg/kg.[2] Doses are than increased to 3 mg/kg.[2]

History

Xenpozyme was the first ASMD-specific treatment.[1]

Society and culture

Olipudase alfa is the international nonproprietary name (INN).[5]

Legal status

Olipudase alfa was approved for medical use in Japan in March 2022.[6]

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.[1] Xenpozyme was reviewed under the accelerated assessment program of the European Medicines Agency (EMA).[1] The applicant for this medicinal product is Genzyme Europe BV.[1] Olipudase alfa was approved for medical use in the European Union in June 2022.[1]

References

  1. ↑ 1.00 1.01 1.02 1.03 1.04 1.05 1.06 1.07 1.08 1.09 1.10 1.11 1.12 1.13 1.14 1.15 "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.
  2. ↑ 2.00 2.01 2.02 2.03 2.04 2.05 2.06 2.07 2.08 2.09 2.10 2.11 2.12 "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.
  3. ↑ "Olipudase alfa". SPS - Specialist Pharmacy Service. 11 January 2016. Archived from the original on 2 July 2022. Retrieved 13 December 2022.
  4. ↑ "Xenpozyme Prices, Coupons, Copay & Patient Assistance". Drugs.com. Retrieved 13 December 2022.
  5. ↑ World Health Organization (2015). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 73". WHO Drug Information. 29 (1). hdl:10665/331088.
  6. ↑ "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.

External links

Identifiers:
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