Alglucosidase alfa explained

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).[1] 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]

It was approved for medical use in the United States in April 2006, as Myozyme[3] and in May 2010, as Lumizyme.[4]

Medical uses

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

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.[5]

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).[6]

In 2015, alglucosidase alfa was ranked the costliest drug per patient, with an average charge of US$630,159.[7]

Notes and References

  1. Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL, Leslie N, Levine J, Spencer C, McDonald M, Li J, Dumontier J, Halberthal M, Chien YH, Hopkin R, Vijayaraghavan S, Gruskin D, Bartholomew D, van der Ploeg A, Clancy JP, Parini R, Morin G, Beck M, De la Gastine GS, Jokic M, Thurberg B, Richards S, Bali D, Davison M, Worden MA, Chen YT, Wraith JE . Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease . Neurology . 68 . 2 . 99–109 . January 2007 . 17151339 . 10.1212/01.wnl.0000251268.41188.04 . free .
  2. Web site: FDA Approves First Treatment for Pompe Disease . FDA . 28 April 2006 . https://web.archive.org/web/20060519213752/http://www.fda.gov/bbs/topics/NEWS/2006/NEW01365.html . dead . 19 May 2006 . 7 July 2008 .
  3. Web site: Drug Approval Package: Myozyme (Alglucosidase Alfa) NDA #125141 . U.S. Food and Drug Administration . 14 August 2020 . 12 April 2021 . https://web.archive.org/web/20210412113934/https://www.accessdata.fda.gov/drugsatfda_docs/nda/2006/125141s000_MyozymeTOC.cfm . live .
  4. Web site: Drug Approval Package: Lumizyme (alglucosidase alfa) NDA #125291 . U.S. Food and Drug Administration . 14 August 2020 . 30 November 2020 . https://web.archive.org/web/20201130024110/https://www.accessdata.fda.gov/drugsatfda_docs/nda/2010/125291s000TOC.cfm . live .
  5. Web site: FDA expands approval of drug to treat Pompe disease to patients of all ages; removes risk mitigation strategy requirements . FDA . 14 August 2014 . https://web.archive.org/web/20140803220414/http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm407563.htm . dead . 3 August 2014 .
  6. News: Geeta . Anand . As Costs Rise, New Medicines Face Pushback . The Wall Street Journal . 18 September 2007 . 7 July 2008 . 21 April 2019 . https://web.archive.org/web/20190421234510/https://www.wsj.com/articles/SB119007210553130427 . live .
  7. Web site: Magellan Rx Management Medical Pharmacy Trend Report . https://web.archive.org/web/20190329161935/https://www1.magellanrx.com/media/604882/2016mrxtrendreport_final.pdf . 29 March 2019 . 2016 . 7th .

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