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 a recombinant human enzyme presented as a 5 mg/mL solution for injection for intravenous use. It treats Pompe disease, including infantile‑onset and late‑onset forms, by replacing deficient acid alpha‑glucosidase activity.

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

Manufacturer / TM Owner

Sanofi B.V.

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Indications & Clinical Uses

Myozyme is used in enzyme replacement therapy for glycogen storage disease type II (Pompe disease).

  • Infantile‑onset Pompe disease: improves survival and motor development by providing functional acid alpha‑glucosidase.
  • Late‑onset Pompe disease: slows progression of respiratory and skeletal muscle weakness through glycogen clearance.
  • Acid alpha‑glucosidase deficiency (Pompe disease): reduces lysosomal glycogen accumulation, helping maintain organ function.

How It Works

  • Alglucosidase alfa is a recombinant form of human acid alpha‑glucosidase that is administered intravenously. The enzyme is taken up by cells via the mannose‑6‑phosphate receptor pathway and delivered to lysosomes, where it hydrolyzes accumulated glycogen into glucose. Restoring this enzymatic activity decreases lysosomal glycogen storage, thereby improving muscle and cardiac function in patients with Pompe disease.

Side Effects

Myozyme may be associated with a range of adverse reactions observed during clinical use.

Common Side Effects

  • Infusion‑related reactions such as fever, chills, or flushing.
  • Headache.
  • Nausea or vomiting.
  • Rash or pruritus.

Serious or Rare Side Effects

  • Anaphylaxis or severe hypersensitivity reactions.
  • Respiratory distress or bronchospasm.
  • Cardiac arrhythmias.
  • Elevated liver enzymes or thrombocytopenia.

Precautions & Warnings

When using Myozyme, several precautions should be observed to ensure patient safety.

  • Cardiac monitoring: assess baseline and ongoing cardiac function due to potential arrhythmias.
  • Respiratory assessment: evaluate pulmonary status before and during infusion.
  • Antibody testing: check for anti‑drug antibodies that may affect efficacy.
  • Dose adjustment: consider renal or hepatic impairment when determining infusion rate.
  • Hypersensitivity: discontinue infusion if severe allergic reaction occurs.
  • Store at 2‑8 °C: keep refrigerated, protect from light, do not freeze or shake.

Avoid Interactions

Myozyme can interact with other medicines, requiring attention to avoid reduced efficacy or increased toxicity.

Major Interactions (Avoid)

  • Live vaccines: concurrent administration may increase risk of infection.
  • Other enzyme replacement therapies: may compete for cellular uptake pathways.
  • High‑dose immunosuppressants: can diminish immune response to the enzyme.

Moderate Interactions (Monitor Closely)

  • Antihistamines: may mask early signs of infusion reactions.
  • Beta‑blockers: can blunt heart rate changes used to monitor infusion safety.
  • ACE inhibitors or ARBs: monitor blood pressure during infusion.

Frequently Asked Questions

Myozyme is prescribed for enzyme replacement therapy in patients with Pompe disease, a rare genetic disorder caused by deficiency of the enzyme acid alpha‑glucosidase. It is indicated for both infantile‑onset and late‑onset forms of the disease to improve muscle and cardiac function.

Alglucosidase alfa is a recombinant version of the missing human enzyme acid alpha‑glucosidase. After intravenous infusion, it is taken up by cells via the mannose‑6‑phosphate receptor and delivered to lysosomes, where it breaks down accumulated glycogen into glucose, reducing cellular storage and improving organ performance.

The dosage of Myozyme is individualized by the prescribing physician based on factors such as the patient’s body weight, disease severity, age, and clinical response. The prescriber decides the infusion rate and total amount required for each treatment cycle.

Safety data for Myozyme in pregnancy and lactation are limited, and the product is classified as having an unknown pregnancy category. It should only be used when the potential benefit to the mother outweighs any possible risk to the fetus or infant.

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

The primary alternative to Myozyme is avalglucosidase alfa (Lumizyme), another recombinant acid alpha‑glucosidase. Both provide enzyme replacement, but they differ in molecular structure, dosing schedules, and regulatory approvals. Clinical choice depends on factors such as patient response, immunogenicity risk, and regional availability.

Myozyme must be stored refrigerated at 2‑8 °C. It should be kept in its original packaging, protected from light, and must never be frozen or shaken. If the product reaches room temperature, it should be used promptly and not re‑refrigerated.

Myozyme is given as an intravenous infusion. The infusion is typically administered over several hours under medical supervision, with vital signs monitored before, during, and after the infusion to detect any adverse reactions.

The most serious risks include severe hypersensitivity reactions such as anaphylaxis, respiratory distress or bronchospasm, cardiac arrhythmias, and significant liver enzyme elevations or thrombocytopenia. Immediate medical attention is required if any of these events occur.

Myozyme is contraindicated in individuals with known hypersensitivity to alglucosidase alfa or any of its excipients. Patients with uncontrolled severe cardiac disease or those who have experienced a serious infusion‑related reaction should also avoid treatment until evaluated by a specialist.

Published by

GNH India Pharmaceuticals Limited

Licensed pharmaceutical supplier

Last updated:

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