Alglucosidase alfa



Drug updated on 4/17/2024

Dosage FormInjection (intravenous; 5mg/mL)
Drug ClassLysosomal glycogen-specific enzymes
Ongoing and
Completed Studies


  • Indicated for use in patients with Pompe disease (GAA deficiency).

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  • Alglucosidase Alfa (Myozyme) is indicated for use in patients with Pompe disease, showing significant improvements in left ventricular mass, time to start ventilation, and survival when compared to the natural history of Pompe Disease or placebo controls. However, no significant improvements were observed in myocardial function and psychomotor development post-enzyme replacement therapy.
  • The effectiveness and safety of Alglucosidase Alfa for treating both infantile-onset Pompe Disease (IOPD) and late-onset Pompe Disease (LOPD) were evaluated in three systematic review studies.
  • For patients with LOPD specifically, treatment with Alglucosidase Alfa has been associated with improvements in the distance walked during a 6-minute walking test, the physical domain of the SF-36 quality of life questionnaire results, as well as time on ventilation. No notable differences were found in functional vital capacity or total SF-36 quality of life score.
  • Across both IOPD and LOPD populations treated with Alglucosidase Alfa, there have been mostly mild adverse events reported after enzyme replacement therapy, which suggests a favorable safety profile for Myozyme.
  • The presence of anti-rhGAA antibody titers may affect treatment response among adults suffering from LOPD; while most high titer cases did not show a clear negative impact on treatment response, some isolated reports suggest interference by sustained neutralizing antibodies. This underscores the importance of monitoring immune responses during individualized treatment planning.
  • In comparison to natural disease progression or placebo control, especially among IOPD patients, Myozyme’s effectiveness stands out, although further research is crucial for refining understanding about long-term outcomes, including how it compares against other treatments, particularly concerning muscle function improvement and quality-of-life enhancements.