BLAINJECTIONINJECTABLEPriority Review
Approved
Dec 2015
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
6
Data completeness
4/8 — Partial
Priority Review
KANUMA (sebelipase alfa) by AstraZeneca is (lal) enzyme. First approved in 2015.
Drug data last refreshed 15h ago · AI intelligence enriched 1mo ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
KANUMA (sebelipase alfa) is a recombinant lysosomal acid lipase (LAL) enzyme replacement therapy administered by intravenous injection. It treats lysosomal acid lipase deficiency (LAL-D), a rare genetic disorder causing progressive lipid accumulation in organs including the liver, spleen, and blood vessels. The drug works by catalyzing the breakdown of cholesteryl esters and triglycerides within lysosomes, restoring normal lipid metabolism.
Peak
Peak-stage orphan therapy with established market presence; commercial team likely stable in size with focus on specialist and hospital channel maintenance.
Mechanism of Action
(LAL) enzyme. The primary site of action of the LAL enzyme is the lysosome, where the enzyme normally causes the breakdown of lipid particles, including LDL-c and triglycerides. Deficient LAL enzyme activity results in progressive complications due to the lysosomal accumulation of cholesteryl…
Pharmacologic Class:
Hydrolytic Lysosomal Cholesteryl Ester-specific Enzyme
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (6)
Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency
Started Jun 2014
31 enrolled
Lysosomal Acid Lipase Deficiency
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
Started Jun 2014
10 enrolled
Lysosomal Acid Lipase Deficiency
Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency
Started Dec 2011
8 enrolled
Cholesterol Ester Storage Disease (CESD)Lysosomal Acid Lipase DeficiencyLAL-Deficiency
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
Started May 2011
9 enrolled
Lysosomal Acid Lipase DeficiencyWolman Disease
Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency
Started Apr 2011
9 enrolled
Cholesterol Ester Storage Disease(CESD)Lysosomal Acid Lipase DeficiencyLAL-Deficiency
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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.