ELELYSO
ELELYSO (taliglucerase alfa) is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for the treatment of Type 1 Gaucher disease. It is approved for use in pediatric and adult patients aged four years and older with a confirmed diagnosis. As an enzyme replacement therapy, it addresses the enzymatic deficiency characteristic of this autosomal recessive disorder.
How ELELYSO Works
Type 1 Gaucher disease is caused by a deficiency in the enzyme glucocerebrosidase, which leads to the accumulation of glucocerebroside in the liver, spleen, and bone marrow. ELELYSO acts as a recombinant analog of this enzyme, catalyzing the conversion of the accumulated substrate into glucose and ceramide. The drug enters cellular lysosomes by binding its mannose oligosaccharide chains to specific mannose receptors on the cell surface. This internalization reduces the amount of stored glucocerebroside within the lysosomal compartment of macrophages.
Details
- Status
- Prescription
- First Approved
- 2012-05-01
- Routes
- INTRAVENOUS
- Dosage Forms
- POWDER
ELELYSO Approval History
What ELELYSO Treats
1 indicationsELELYSO is approved for 1 conditions since its original approval in 2012. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Gaucher Disease
ELELYSO Boxed Warning
HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life‑threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate ELELYSO in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reactio...
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life‑threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate ELELYSO in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue ELELYSO and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life‑threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1) ] . WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS See full prescribing information for complete boxed warning. • Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. ( 5.1 ) • Initiate ELELYSO in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. ( 5.1 ) • If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue ELELYSO and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 )
Drugs Similar to ELELYSO
FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
ELELYSO FDA Label Details
ProIndications & Usage
FDA Label (PDF)ELELYSO is indicated for the treatment of patients 4 years of age and older with a confirmed diagnosis of Type 1 Gaucher disease. ELELYSO is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for the treatment of patients 4 years and older with a confirmed diagnosis of Type 1 Gaucher disease .
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life‑threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration...
Want competitive intelligence?
See who's developing similar drugs and track their progress
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.