Alglucosidase alfa

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Alglucosidase alfa
Clinical data
Trade names Myozyme, Lumizyme, others
AHFS/Drugs.com Monograph
License data
Routes of
administration 		Intravenous [1] [2]
ATC code
Legal status
Legal status
Identifiers
  • Human glucosidase, prepro-α-[199-arginine,223-histidine] [5]
CAS Number
DrugBank
ChemSpider
  • none
UNII
KEGG
Chemical and physical data
Formula C4758H7262N1274O1369S35 [5]
Molar mass 105338 g·mol−1 [5]
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Alglucosidase alfa, sold under the brand name Myozyme among others, is an enzyme replacement therapy (ERT) orphan drug for treatment of Pompe disease (Glycogen storage disease type II), a rare lysosomal storage disorder (LSD). [6] Chemically, the drug is an analog of the enzyme that is deficient in patients affected by Pompe disease, alpha-glucosidase. It is the first drug available to treat this disease. [2]

Contents

It was approved for medical use in the United States in April 2006, as Myozyme [7] and in May 2010, as Lumizyme. [8]

Medical uses

Alglucosidase alfa is indicated for people with Pompe disease (GAA deficiency). [1]

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. [9]

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. [2]

Society and culture

Economics

Some American health plans have refused to subsidize alglucosidase alfa for adults because it lacks approval for treatment in adults, as well as its high cost (US$ 300,000 per year for life). [10]

In 2015, alglucosidase alfa was ranked the costliest drug per patient, with an average charge of US$630,159. [11]

Related Research Articles

<span class="mw-page-title-main">Glycogen storage disease type II</span> Medical condition

Glycogen storage disease type II(GSD-II), also called Pompe disease, and formerly known as GSD-IIa or Limb–girdle muscular dystrophy2V, is an autosomal recessive metabolic disorder which damages muscle and nerve cells throughout the body. It is caused by an accumulation of glycogen in the lysosome due to deficiency of the lysosomal acid alpha-glucosidase enzyme (GAA). The inability to breakdown glycogen within the lysosomes of cells leads to progressive muscle weakness throughout the body and affects various body tissues, particularly in the heart, skeletal muscles, liver and the nervous system.

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References

  1. 1 2 3 "Lumizyme- alglucosidase alfa injection, powder, for solution". DailyMed. 22 February 2020. Archived from the original on 25 March 2021. Retrieved 14 August 2020.
  2. 1 2 3 "FDA Approves First Treatment for Pompe Disease". FDA. 28 April 2006. Archived from the original on 19 May 2006. Retrieved 7 July 2008.
  3. ↑ "Myozyme (alglucosidase alfa) Injectable for intravenous infusion Initial U.S. Approval: 2006". DailyMed. Archived from the original on 18 December 2022. Retrieved 18 December 2022.
  4. ↑ "Myozyme EPAR". European Medicines Agency. 14 February 2001. Archived from the original on 28 December 2021. Retrieved 15 May 2024.
  5. 1 2 3 American Medical Association (USAN). "Alglucosidase alfa". Statement on a Nonproprietary Name Adopted by the USAN Council. Archived from the original (Microsoft Word) on 12 February 2012. Retrieved 18 December 2007.
  6. ↑ Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL, et al. (January 2007). "Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease". Neurology. 68 (2): 99–109. doi: 10.1212/01.wnl.0000251268.41188.04 . PMID   17151339.
  7. ↑ "Drug Approval Package: Myozyme (Alglucosidase Alfa) NDA #125141". U.S. Food and Drug Administration. Archived from the original on 12 April 2021. Retrieved 14 August 2020.
  8. ↑ "Drug Approval Package: Lumizyme (alglucosidase alfa) NDA #125291". U.S. Food and Drug Administration. Archived from the original on 30 November 2020. Retrieved 14 August 2020.
  9. ↑ "FDA expands approval of drug to treat Pompe disease to patients of all ages; removes risk mitigation strategy requirements". FDA. 14 August 2014. Archived from the original on 3 August 2014.
  10. ↑ Anand G (18 September 2007). "As Costs Rise, New Medicines Face Pushback". The Wall Street Journal. Archived from the original on 21 April 2019. Retrieved 7 July 2008.
  11. ↑ "Magellan Rx Management Medical Pharmacy Trend Report" (PDF) (7th ed.). 2016. Archived from the original (PDF) on 29 March 2019.
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