KANUMA
Kanuma (sebelipase alfa) is a hydrolytic lysosomal enzyme indicated for the treatment of patients diagnosed with Lysosomal Acid Lipase (LAL) deficiency. This autosomal recessive disorder is characterized by a genetic defect that results in a marked decrease or loss of the LAL enzyme. The therapy addresses the progressive complications caused by the accumulation of cholesteryl esters and triglycerides in organs such as the liver, spleen, and intestine.
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.
Details
- Status
- Prescription
- First Approved
- 2015-12-08
- 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 )
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
ProIndications & Usage
FDA Label (PDF)KANUMA ® is indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency. KANUMA ® is a hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) 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.