Alglucosidase alfa

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Alglucosidase alfa
Names
Trade namesMyozyme, Lumizyme, others
  • Human glucosidase, prepro-α-[199-arginine,223-histidine] [1]
Clinical data
Drug classEnzyme[2]
Main usesPompe disease[3]
Side effectsAllergic reactions, fever, headache, sweating, nausea, tiredness, muscle pain[4]
Routes of
use
Intravenous[4][5]
Typical dose20 mg/kg[6]
External links
AHFS/Drugs.comMonograph
US NLMAlglucosidase alfa
Legal
License data
Legal status
Chemical and physical data
FormulaC4758H7262N1274O1369S35[1]
Molar mass105338 g·mol−1[1]
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Alglucosidase alfa, sold under the brand name Myozyme among others, is a medication to treat Pompe disease (glycogen storage disease type II).[3] It is given by gradual injection into a vein.[6]

Common side effects include allergic reactions, fever, headache, sweating, nausea, tiredness, and muscle pain.[4] Other side effects may include anaphylaxis.[4] It is an enzyme that replaces the missing alpha-glucosidase.[2]

Alglucosidase alfa was approved for medical use in the United States and Europe in 2006.[3][2] In the United Kingdom 50 mg costs the NHS about £360 as of 2021.[6] This amount in the United States is about 900 USD.[7]

Medical uses

Alglucosidase alfa is indicated for people with Pompe disease (GAA deficiency).[4]

In 2014 the U.S. Food and Drug Administration announced the approval of alglucosidase alfa for treatment of people with infantile-onset Pompe disease, including people who are less than eight years of age. In addition, the Risk Evaluation and Mitigation Strategy (REMS) is being eliminated.[8]

Dosage

It is given at a dose of 20 mg/kg every two weeks.[6]

Side effects

Common observed adverse reactions to alglucosidase alfa treatment are pneumonia, respiratory complications, infections and fever. More serious reactions reported include heart and lung failure and allergic shock. Myozyme boxes carry warnings regarding the possibility of life-threatening allergic response.[5]

History

It is the first drug available to treat this disease.[5]

Cost

Some health plans have refused to subsidize Myozyme for adults because it lacks approval for treatment in adults, as well as its high cost (US$300,000 per year for life).[9]

In 2015, Lumizyme was ranked the costliest drug per patient, with an average charge of $630,159.[10]

References

  1. 1.0 1.1 1.2 American Medical Association (USAN). "Alglucosidase alfa" (Microsoft Word). Statement on a Nonproprietary Name Adopted by the USAN Council. Retrieved 18 December 2007.
  2. 2.0 2.1 2.2 "Myozyme". Retrieved 13 January 2022.
  3. 3.0 3.1 3.2 "Alglucosidase Alfa Monograph for Professionals". Drugs.com. Retrieved 13 January 2022.
  4. 4.0 4.1 4.2 4.3 4.4 4.5 "Lumizyme- alglucosidase alfa injection, powder, for solution". DailyMed. 22 February 2020. Retrieved 14 August 2020.
  5. 5.0 5.1 5.2 "FDA Approves First Treatment for Pompe Disease". FDA. 2006-04-28. Retrieved 2008-07-07.[permanent dead link]
  6. 6.0 6.1 6.2 6.3 BNF 81: March-September 2021. BMJ Group and the Pharmaceutical Press. 2021. p. 1117. ISBN 978-0857114105.
  7. "Lumizyme Prices, Coupons & Patient Assistance Programs". Drugs.com. Retrieved 13 January 2022.
  8. "FDA expands approval of drug to treat Pompe disease to patients of all ages; removes risk mitigation strategy requirements". FDA. 2014-08-14. Archived from the original on 2014-08-03.
  9. Anand G (2007-09-18). "As Costs Rise, New Medicines Face Pushback". The Wall Street Journal. Dow Jones & Company. Retrieved 2008-07-07.
  10. "Magellan Rx Management Medical Pharmacy Trend Report" (PDF) (7th ed.). 2016. Archived from the original (PDF) on 29 March 2019.

External links

Identifiers: