Product image of Myozyme (Alglucosidase Alfa) supplied by GNH India

Myozyme

Active Ingredient:
Alglucosidase Alfa
Origin:
EU

Myozyme (Alglucosidase Alfa)

Alglucosidase alfa, the active ingredient in Myozyme, is an enzyme replacement therapy presented as a 20 mg per vial (5 mg/mL) lyophilized powder for solution for injection for intravenous use. It replaces the deficient acid alpha‑glucosidase enzyme in patients with Pompe disease (acid alpha‑glucosidase deficiency).

GNH India supplies Myozyme as a licensed, GDP‑compliant international pharmaceutical supplier serving hospitals, transplant centres, pharmacies and clinics worldwide.

Manufacturer / TM Owner

Sanofi B.V.

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Indications & Clinical Uses

Myozyme is used in the management of Pompe disease, a lysosomal storage disorder caused by acid alpha‑glucosidase deficiency.

  • Infantile‑onset Pompe disease: improves survival and motor development.
  • Late‑onset Pompe disease: stabilizes respiratory function and muscle strength.
  • Adult Pompe disease: reduces disease progression and supports daily activities.

How It Works

  • Alglucosidase alfa is a recombinant human acid alpha‑glucosidase enzyme that is taken up by cells via the mannose‑6‑phosphate receptor pathway and delivered to lysosomes. Once inside, it hydrolyzes accumulated glycogen, restoring normal glycogen turnover and reducing lysosomal storage. By supplementing the missing endogenous enzyme, it addresses the underlying metabolic defect of Pompe disease.

Side Effects

Myozyme may be associated with infusion‑related reactions and other adverse events.

Common Side Effects

  • Fever: transient elevation of body temperature during or after infusion.
  • Nausea: mild to moderate sensation of stomach upset.
  • Headache: occasional mild headache following administration.
  • Rash: localized skin redness or itching at the infusion site.

Serious or Rare Side Effects

  • Anaphylaxis: severe allergic reaction requiring immediate medical attention.
  • Hypotension: sudden drop in blood pressure during infusion.
  • Cardiac arrhythmia: irregular heart rhythm observed in some patients.
  • Pulmonary edema: fluid accumulation in the lungs presenting with breathing difficulty.

Precautions & Warnings

Before initiating Myozyme therapy, several precautions should be considered to ensure patient safety.

  • Renal function: assess baseline kidney status and monitor during treatment.
  • Cardiac status: evaluate heart function because patients may have underlying cardiomyopathy.
  • Infusion rate: start at a slow rate and adjust to minimize infusion reactions.
  • Immunogenicity: monitor for development of anti‑drug antibodies that could reduce efficacy.
  • Pregnancy: use only if the potential benefit justifies the potential risk to the fetus.
  • Store at 2‑8 °C: keep refrigerated, protect from light, do not freeze or shake.

Avoid Interactions

Myozyme can interact with other medicines, affecting safety or efficacy.

Major Interactions (Avoid)

  • Concomitant lysosomal enzyme therapies: may increase immunogenic risk.
  • Live vaccines: may have reduced effectiveness due to immune modulation.

Moderate Interactions (Monitor Closely)

  • Antihistamines: can mask early signs of infusion reactions.
  • Corticosteroids: may alter immune response and affect antibody formation.
  • Other intravenous biologics: schedule to avoid overlapping infusion times.

Frequently Asked Questions

Myozyme is indicated for the treatment of Pompe disease, also known as acid alpha‑glucosidase deficiency. It is used in infantile‑onset, late‑onset, and adult forms of the disease to replace the missing enzyme and improve clinical outcomes.

Alglucosidase alfa is a recombinant form of the human acid alpha‑glucosidase enzyme. After intravenous infusion, it is taken up by cells via mannose‑6‑phosphate receptors and delivered to lysosomes, where it hydrolyzes accumulated glycogen, restoring normal glycogen breakdown and reducing cellular storage.

The dose of Myozyme is individualized. The prescribing clinician determines the amount based on factors such as the patient’s body weight, age, disease severity, and clinical response. No fixed regimen is provided here; dosing decisions are made by the treating physician.

Myozyme is classified as Pregnancy Category B, indicating that animal studies have not shown risk but there are limited human data. It should be used during pregnancy only if the potential benefit justifies any potential risk to the fetus. Breastfeeding decisions should be made in consultation with a healthcare professional.

To order Myozyme, submit an enquiry on the product page at gnhindia.com, specifying the required quantity. GNH India supplies hospitals, pharmacies and procurement teams worldwide, verifies trade or institutional credentials, and then provides pricing, availability, cold‑chain shipping details and any necessary import documentation.

Myozyme (alglucosidase alfa) is one of the approved enzyme replacement therapies for Pompe disease. An alternative product, Lumizyme (also alglucosidase alfa but marketed under a different brand), has a similar mechanism and indication. Differences may lie in formulation, packaging, and regional regulatory status, but both aim to replace the deficient enzyme.

Myozyme should be stored at 2‑8 °C (refrigerated). Keep the vials in the original packaging, protect them from light, and avoid freezing or shaking. Once reconstituted, the solution should be used promptly according to the product’s handling instructions.

Myozyme is supplied as a lyophilized powder that must be reconstituted with the appropriate diluent to form a solution for injection. The solution is then administered intravenously, typically as an infusion over a period determined by the prescribing clinician.

Serious risks include anaphylaxis, severe hypotension, cardiac arrhythmias, and pulmonary edema. These events are rare but require immediate medical attention. Patients should be monitored closely during and after the infusion for any signs of these reactions.

Myozyme is contraindicated in individuals with a known hypersensitivity to alglucosidase alfa or any of the product’s excipients. Caution is also advised for patients with severe renal impairment or uncontrolled cardiac disease, and the decision to treat should be made by a qualified healthcare professional.

Published by

GNH India Pharmaceuticals Limited

Licensed pharmaceutical supplier

Last updated:

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