ELELYSO (taliglucerase alfa)
ELELYSO is indicated for the treatment of Gaucher Disease.
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.
Development Insights
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 )
ELELYSO Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
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Clinical Trial Registry
6 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT04002830 results posted | WI224302 | Ph 4 | completed | A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease |
| NCT01422187 results posted | PB-06-007 | Ph 3 | completed | A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease |
| NCT01132690 results posted | PB-06-005 | Ph 4 | completed | A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease |
| NCT00712348 results posted | PB-06-002 | Ph 3 | completed | Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase |
| NCT00705939 results posted | PB-06-003 | Ph 3 | completed | Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial |
| NCT01411228 results posted | PB-06-006 | Ph 3 | completed | A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease |
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
Indications & Usage
FDA Label (PDF)ELELYSO is indicated for the treatment of 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...
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment