BLAINTRAVENOUSPOWDER
Approved
May 2012
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
6
Data completeness
5/8 — Partial
ELELYSO (taliglucerase alfa) by Pfizer is gene, which results in a reduced activity of the lysosomal enzyme glucocerebrosidase. First approved in 2012.
Drug data last refreshed 11h ago · AI intelligence enriched 1w 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 deficiency of glucocerebrosidase. It works by catalyzing the hydrolysis of accumulated glucocerebroside in lysosomes, reducing substrate accumulation in macrophages, liver, spleen, and bone marrow. The drug is administered intravenously as a powder formulation.
$3MPart D
PeakPeak lifecycle stage with modest Part D utilization suggests stable but niche orphan market positioning with limited team expansion.
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
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (6)
A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease
Started Nov 2020
14 enrolled
Gaucher Disease, Type 3
Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease
Started Jul 2019
0Type 1 Gaucher Disease
Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs
Started Jan 2017
30 enrolled
Gaucher Disease, Type 1
A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Started Sep 2011
15 enrolled
Gaucher Disease
A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
Started Aug 2011
19 enrolled
Gaucher Disease
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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.