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 18h ago · AI intelligence enriched 3w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
NEXVIAZYME (avalglucosidase alfa-ngpt) is a recombinant hydrolytic lysosomal enzyme approved by the FDA on August 6, 2021, for the treatment of Pompe disease, a rare genetic disorder characterized by glycogen accumulation in muscles and organs. It functions as an enzyme replacement therapy that breaks down glycogen in lysosomes, addressing the underlying pathophysiology of this life-threatening condition. NEXVIAZYME represents an improved formulation designed to enhance enzyme uptake and efficacy compared to earlier enzyme replacement options in this ultra-rare disease space.
$0.0BPart D
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
NEXVIAZYME creates specialized roles in rare disease management including product managers, medical science liaisons with rare disease expertise, and specialized sales representatives trained in ultra-rare genetic disorders and hospital/specialty pharmacy channels. Success in this role requires deep knowledge of Pompe disease pathophysiology, payer negotiation skills for high-cost therapies, and ability to identify and engage with small patient populations. Currently, 0 open positions are linked to this product in available job data.
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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.