BLAINTRAVENOUSPOWDER
Approved
May 2012
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
6
Data completeness
6/8 — Strong
ELELYSO (taliglucerase alfa) by Pfizer is gene, which results in a reduced activity of the lysosomal enzyme glucocerebrosidase. Approved for gaucher disease. First approved in 2012.
Drug data last refreshed 21h ago · AI intelligence enriched 2w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
ELELYSO (taliglucerase alfa) is a recombinant enzyme replacement therapy for Gaucher disease, a rare lysosomal storage disorder caused by glucocerebrosidase deficiency. It catalyzes the hydrolysis of accumulated glucocerebroside in macrophages (Gaucher cells) to glucose and ceramide, reducing pathogenic substrate accumulation in the liver, spleen, and bone marrow. The drug is administered intravenously and works by binding mannose oligosaccharide chains to cell surface receptors, enabling cellular uptake and lysosomal targeting.
$0.003BPart D
PeakProduct is at peak maturity with modest Part D penetration (64 claims in 2023), indicating a stable but niche market with limited team expansion opportunity.
Mechanism of Action
gene, which results in a reduced activity of the lysosomal enzyme glucocerebrosidase. Glucocerebrosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide. The enzymatic deficiency results in accumulation of substrate glucocerebroside primarily in the lysosomal…
Pharmacologic Class:
Hydrolytic Lysosomal Glucocerebroside-specific Enzyme
Indications (1)
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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.