Sebelipasa Alfa

Sebelipasa Alfa Uses, Dosage, Side Effects, Food Interaction and all others data.

Sebelipasa Alfa is a recombinant form of the enzyme lysosomal acid lipase (LAL) approved for the treatment of lysosomal acid lipase deficiency (LAL-D). The amino acid sequence for sebelipase alfa is the same as the amino acid sequence for human LAL. Sebelipasa Alfa is an orphan drug which is expected to cost about $310,000 for annual treatment in the United States. Sebelipasa Alfa is marketed under the brand name Kanuma™ by Alexion Pharmaceuticals, Inc.

Sebelipasa Alfa
Uses
Dosage
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Uses during Pregnancy
Uses during Breastfeeding
Accute Overdose
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Trade Name	Sebelipasa Alfa
Generic	Sebelipase alfa
Sebelipase alfa Other Names	SBC-102, Sebelipasa alfa, Sebelipase alfa
Type
Formula	C₁₉₆₈H₂₉₄₅N₅₀₇O₅₅₁S₁₅
Weight	55000.0 Da
Groups	Approved, Investigational
Therapeutic Class
Manufacturer
Available Country
Last Updated:	September 19, 2023 at 7:00 am

Uses

Sebelipasa Alfa is a recombinant lysosomal acid lipase used to treat lysosomal acid lipase deficiency.

Sebelipasa Alfa 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.

Sebelipasa Alfa is also used to associated treatment for these conditions: Lysosomal Acid Lipase Deficiency

How Sebelipasa Alfa works

LAL deficiency is an autosomal recessive lysosomal storage disorder characterized by a genetic defect resulting in a marked decrease or loss in activity of the lysosomal acid lipase (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. Deficient LAL enzyme activity results in progressive complications due to the lysosomal accumulation of cholesteryl esters and triglycerides in multiple organs, including the liver, spleen, intestine, and the walls of blood vessels. The resulting lipid accumulation in the liver may lead to increased liver fat content and progression of liver disease, including fibrosis and cirrhosis. Lipid accumulation in the intestinal wall leads to malabsorption and growth failure. In parallel, dyslipidemia due to impaired degradation of lysosomal lipid is common with elevated LDL-c and triglycerides and low HDL-cholesterol (HDL-c). Sebelipasa Alfa binds to cell surface receptors via glycans expressed on the protein and is subsequently internalized into lysosomes. Sebelipasa Alfa catalyzes the lysosomal hydrolysis of cholesteryl esters and triglycerides to free cholesterol, glycerol and free fatty acids.