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sebelipase alfa
- Development stage
- Approved
- Lead developer
- Alexion Pharmaceuticals
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Administration
- Intravenous
01
Overview
Sebelipase alfa is a recombinant form of the human enzyme lysosomal acid lipase (LAL), produced using genetically modified chickens to express the enzyme in egg whites. It is used as an enzyme replacement therapy for patients with lysosomal acid lipase deficiency (LAL-D), a rare, life-threatening genetic disorder characterized by the accumulation of cholesteryl esters and triglycerides in various tissues due to deficient LAL activity. The drug works by replacing the missing or deficient LAL enzyme, thereby reducing lipid accumulation and improving liver function and lipid profiles. Sebelipase alfa is administered via intravenous infusion and has demonstrated efficacy in both pediatric and adult populations[1][2][4][8].
- Brand names
- Kanuma
- Other names
- recombinant human lysosomal acid lipaserhLAL
02
Targets
IGF2R (Cation-independent mannose-6-phosphate receptor)
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