KANUMA (sebelipase alfa)
KANUMA is indicated for the treatment of Lysosomal Acid Lipase Deficiency.
How KANUMA Works
Sebelipase alfa binds to cell surface receptors and is internalized into the lysosomes of affected cells. Once inside, the enzyme catalyzes the hydrolysis of accumulated cholesteryl esters and triglycerides into free cholesterol, glycerol, and free fatty acids. By replacing the missing enzyme activity, the drug facilitates the breakdown of lipid particles and helps prevent their accumulation in multiple organ systems and blood vessel walls.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2015-12-08
- Patent Cliff
- 2022
- Routes
- INJECTION
- Dosage Forms
- INJECTABLE
KANUMA Approval History
What KANUMA Treats
1 indicationsKANUMA is approved for 1 conditions since its original approval in 2015. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Lysosomal Acid Lipase Deficiency
KANUMA 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 KANUMA in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction...
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 KANUMA 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 KANUMA 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 KANUMA in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation. ( 5.1 ) If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue KANUMA and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 )
KANUMA Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
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.
Clinical Trial Registry
4 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT01757184 ARISE results posted | LAL-CL02 | Ph 3 | completed | Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency |
| NCT02112994 results posted | LAL-CL06 2011-004287-30 | Ph 2 | completed | Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency |
| NCT02193867 results posted | LAL-CL08 | Ph 2 | terminated | Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency |
| NCT01488097 results posted | LAL-CL04 | Ph 2 | completed | Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency |
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.
KANUMA FDA Label Details
Indications & Usage
FDA Label (PDF)KANUMA is indicated for the treatment of Lysosomal Acid Lipase Deficiency.
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