BLAINJECTIONINJECTABLEPriority Review
Approved
Aug 2021
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
1
Data completeness
5/8 — 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 13h 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 to treat Pompe disease, a rare genetic disorder characterized by glycogen accumulation in muscles and organs. It is an enzyme replacement therapy (ERT) that degrades lysosomal glycogen, restoring cellular function in patients with deficient acid alpha-glucosidase activity. The drug represents a next-generation ERT with improved targeting and pharmacokinetics compared to earlier options.
$22MPart D
PeakDrug is in peak lifecycle stage with modest Part D spending (~$22M in 2023), indicating stable but limited market penetration typical of ultra-rare disease therapies, requiring focused commercial execution in niche neuromuscular channels.
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
Why This Matters for Your Career
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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.