Alglucosidase alfa

Not to be confused with Avalglucosidase alfa.

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]

Alglucosidase alfa

Clinical data
Trade names	Myozyme, Lumizyme, others
AHFS/Drugs.com	Monograph
License data	US DailyMed: Alglucosidase alfa
Routes of administration	Intravenous[1][2]
ATC code	A16AB07 (WHO)
Legal status
Legal status	AU: S4 (Prescription only) US: ℞-only[1][3] EU: Rx-only[4]
Identifiers
IUPAC name Human glucosidase, prepro-α-[199-arginine,223-histidine][5]
CAS Number	420784-05-0 Y
DrugBank	DB01272 Y
ChemSpider	none
UNII	DTI67O9503
KEGG	D03207 N
Chemical and physical data
Formula	C4758H7262N1274O1369S35[5]
Molar mass	105338 g·mol−1[5]
NY (what is this?)  (verify)

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]

References

1. "Lumizyme- alglucosidase alfa injection, powder, for solution". DailyMed. 22 February 2020. Archived from the original on 25 March 2021. Retrieved 14 August 2020.
2. "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. 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.

   • Lay summary in: "Center for Drug Evaluation and Research: Application Number: 125291: Summary Review" (PDF). Food and Drug Administration.
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.