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avalglucosidase alfa-ngpt (Nexviazyme)

Indications: - enzyme replacement therapy for Pompe disease * avalglucosidase alfa-ngpt (Nexviazyme) indicated for late-onset disease >= 1 year of age (FDA-approved) * alglucosidase alfa (Lumizyme) indicated for patients >= 8 years of age Dosage: - IV infusion every 2 weeks Adverse effects: - hypersensitivity reactions including anaphylaxis - infusion-associated reactions - arthralgia, nausea, vomiting, diarrhea - headache, myalgia, paresthesia, dizziness, fatigue - erythema, pruritus, urticaria - persons susceptible to fluid volume overload or with compromised cardiac or respiratory function may be at risk for serious acute cardiorespiratory failure Mechanism of action: - target the mannose-6-phosphate (M6P) receptor

General

metabolic agent (metabolic modifier)

References

  1. Ingram I New Enzyme Therapy OK'd for Pompe Disease. IV treatment improved lung function, 6-minute walk test in later-onset disease. MedPage Today August 6, 2021 https://www.medpagetoday.com/publichealthpolicy/fdageneral/93944
  2. Brooks M FDA Clears New Enzyme Replacement Therapy for Pompe Disease Medscape - Aug 06, 2021. https://www.medscape.com/viewarticle/956179
  3. HIGHLIGHTS OF PRESCRIBING INFORMATION LUMIZYME (alglucosidase alfa). for injection, for intravenous use https://www.accessdata.fda.gov/drugsatfda_docs/label/2010/125291lbl.pdf