|Main uses||Lysosomal acid lipase deficiency (LAL-D)|
|Side effects||Headache, fever, throat pain, nausea, constipation, allergic reactions|
|Typical dose||1 mg/kg|
|US NLM||Sebelipase alfa|
|Elimination half-life||0.1 hours|
Sebelipase alfa, sold under the brand name Kanuma, is a medication used to treat lysosomal acid lipase deficiency (LAL-D). Benefit is clearest in Wolman disease. It is given by injection into a vein.
Common side effects include headache, fever, throat pain, nausea, and constipation. Other side effects may include allergic reactions. It is a recombinant form of the lysosomal acid lipase (LAL) enzyme, which helps to break down lipids.
Sebelipase alfa was approved for medical use in Europe and the United States in 2015. In Canada it cost about 892,000 CAD for an adult per year in 2018. In the United States this amount costs about 970,000 USD per year as of 2021. Its use was rejected by the United Kingdom in 2017.
Sebelipase alfa is indicated for long-term enzyme replacement therapy (ERT) in people of all ages with lysosomal acid lipase (LAL) deficiency.
It is given at a dose of 1 mg/kg every two weeks.
Sebelipase was developed by Synageva that became part of Alexion Pharmaceuticals in 2015. For its production, chickens are genetically modified to produce the recombinant form of LAL (rhLAL) in their egg white. After extraction and purification it becomes available as the medication. On December 8, 2015 the FDA announced that its approval came from two centers: The Center for Drug Evaluation and Research (CDER) approved the human therapeutic application of the medication, while the Center for Veterinary Medicine (CVM) approved the application for a recombinant DNA construct in genetically engineered chicken to produce rhLAL in their egg whites. At the time it gained FDA approval Kanuma was the first only drug manufactured in chicken eggs and intended for use in humans.
Society and culture
According to an estimate by a Barclays analyst in 2015, the drug was expected to be priced at about US$375,000 per year.
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