Sebelipase alfa is a recombinant form of the enzyme lysosomal acid lipase (LAL) that was approved in 2015 under the tradename Kanuma in the US and EU for the treatment of people with lysosomal acid lipase deficiency (LAL-D).
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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.
According to an estimate by a Barclays analyst in 2015, the drug was expected to be priced at about US $375,000 per year.
It is administered once a week via intraveneous infusion in people with rapidly progressing disease in the first six months of life. In people with less aggressive disease it is given every other week.
- "New Drugs Online Report for sebelipase alfa". UK Medicines Information. Retrieved December 10, 2015.
- Sheridan, Cormac (5 February 2016). "FDA approves 'farmaceutical' drug from transgenic chickens". Nature Biotechnology. doi:10.1038/nbt0216-117. Retrieved 15 February 2016.
- "FDA approves first drug to treat a rare enzyme disorder in pediatric and adult patients". FDA. December 8, 2015. Retrieved December 10, 2015.
- Burton, B. K.; et al. (September 10, 2015). "A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency". 373. New England Journal of Medicine: 1010–1020. doi:10.1056/NEJMoa1501365. PMID 26352813.
- Sebelipase alfa Label Last updated Dec 2015. See FDA index page for labels here
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