BLAINJECTIONINJECTABLEPriority Review
Approved
Aug 2021
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
1
Data completeness
4/8 — Partial
Priority Review
NEXVIAZYME (avalglucosidase alfa-ngpt) by Sanofi. Approved for hydrolytic lysosomal glycogen-specific enzyme [epc]. First approved in 2021.
Drug data last refreshed 4h ago · AI intelligence enriched 6d ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
NEXVIAZYME (avalglucosidase alfa-ngpt) is a recombinant hydrolytic lysosomal enzyme approved for Pompe disease, a rare genetic disorder caused by acid alpha-glucosidase (GAA) deficiency. It works by replacing the deficient enzyme to break down glycogen accumulation in muscles and heart. The product represents a next-generation approach to enzyme replacement therapy (ERT) for this ultra-rare lysosomal storage disorder.
$22MPart D
PeakProduct is in peak commercial phase with modest Part D penetration; brand team is focused on market expansion and patient access optimization for rare disease.
Mechanism of Action
Pharmacologic Class:
Hydrolytic Lysosomal Glycogen-specific Enzyme
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (1)
A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II
Started Jan 2003
2 enrolled
Glycogen Storage Disease Type IIPompe DiseaseAcid Maltase Deficiency Disease+1 more
Career Relevance
Working on NEXVIAZYME offers exposure to ultra-rare disease commercialization, specialty pharma field operations, and high-touch patient engagement models. Career growth is driven by deep disease expertise, payer/provider relationships, and clinical outcome storytelling rather than volume-based metrics.
Brand Manager
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.