Alglucosidase alfa

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

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.

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.

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).

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

References

References

1. American Medical Association ([[United States Adopted Name. "Alglucosidase alfa". Statement on a Nonproprietary Name Adopted by the USAN Council.
2. (22 February 2020). "Lumizyme- alglucosidase alfa injection, powder, for solution".
3. "Myozyme (alglucosidase alfa) Injectable for intravenous infusion Initial U.S. Approval: 2006".
4. (14 February 2001). "Myozyme EPAR".
5. (January 2007). "Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease". Neurology.
6. (28 April 2006). "FDA Approves First Treatment for Pompe Disease". FDA.
7. "Drug Approval Package: Myozyme (Alglucosidase Alfa) NDA #125141".
8. (14 August 2014). "FDA expands approval of drug to treat Pompe disease to patients of all ages; removes risk mitigation strategy requirements". FDA.
9. Anand, Geeta. (18 September 2007). "As Costs Rise, New Medicines Face Pushback". The Wall Street Journal.
10. (2016). "Magellan Rx Management Medical Pharmacy Trend Report".