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Nexviazyme FDA Approval History

Last updated by Judith Stewart, BPharm on Aug 9, 2021.

FDA Approved: Yes (First approved August 6, 2021)
Brand name: Nexviazyme
Generic name: avalglucosidase alfa-ngpt
Dosage form: Lyophilized Powder for Injection
Company: Sanofi
Treatment for: Pompe disease

Nexviazyme (avalglucosidase alfa-ngpt) is a hydrolytic lysosomal glycogen-specific enzyme indicated for the treatment of patients with late-onset Pompe disease.

  • Pompe disease is caused by a genetic deficiency of alpha-glucosidase (GAA), which results in build-up of glycogen and subsequent irreversible damage to the skeletal muscles and the muscles that support respiratory function.
  • Nexviazyme is indicated for the treatment of patients one year of age and older with late-onset Pompe disease [lysosomal acid alpha-glucosidase (GAA) deficiency].
  • Nexviazyme works by specifically targeting the mannose-6-phosphate (M6P) receptor, the key pathway for GAA enzyme replacement therapy, to effectively clear glycogen build-up in muscle cells.
  • Nexviazyme is administered via intravenous infusion every two weeks.
  • The Nexviazyme product label carries a boxed warning for hypersensitivity reactions, infusion-associated reactions, and the risk of acute cardiorespiratory failure in susceptible patients.
  • Common adverse reactions include headache, fatigue, diarrhea, nausea, arthralgia, dizziness, myalgia, pruritus, vomiting, dyspnea, erythema, paresthesia and urticaria.

Development timeline for Nexviazyme

Aug  6, 2021Approval FDA Approves Nexviazyme (avalglucosidase alfa-ngpt) for Late-Onset Pompe Disease
Feb  2, 2021Data Presented at WORLDSymposium Reinforces Robust Rare Disease Pipeline and Highlights Additional Clinical Data for Investigational Avalglucosidase Alfa in Pompe Disease
Nov 18, 2020FDA Grants Priority Review for Avalglucosidase Alfa, a Potential New Therapy for Pompe Disease

Further information

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