Sebelipase alfa, sold under the brand name Kanuma, is a recombinant form of the

enzyme An enzyme () is a protein that acts as a biological catalyst by accelerating chemical reactions. The molecules upon which enzymes may act are called substrate (chemistry), substrates, and the enzyme converts the substrates into different mol ...

lysosomal acid lipase (LAL) that is used as a

medication Medication (also called medicament, medicine, pharmaceutical drug, medicinal product, medicinal drug or simply drug) is a drug used to medical diagnosis, diagnose, cure, treat, or preventive medicine, prevent disease. Drug therapy (pharmaco ...

for the treatment of lysosomal acid lipase deficiency (LAL-D). Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. It is administered via intraveneous infusion. It was approved for medical use in the European Union and in the United States in 2015.

Medical uses

Sebelipase alfa is indicated for long-term enzyme replacement therapy (ERT) in people of all ages with lysosomal acid lipase (LAL) deficiency.

History

Sebelipase was developed by Synageva that became part of Alexion Pharmaceuticals in 2015. For its production,

chicken The chicken (''Gallus gallus domesticus'') is a domesticated subspecies of the red junglefowl (''Gallus gallus''), originally native to Southeast Asia. It was first domesticated around 8,000 years ago and is now one of the most common and w ...

s are genetically modified to produce the recombinant form of LAL (rhLAL) in their

egg white Egg white is the clear liquid (also called the albumen or the glair/glaire) contained within an egg. In chickens, it is formed from the layers of secretions of the anterior section of the hen's oviduct during the passage of the egg. It forms a ...

. After extraction and purification it becomes available as the medication. On 8 December 2015 the FDA announced that its approval came from two centers: The

Center for Drug Evaluation and Research The Center for Drug Evaluation and Research (CDER, pronounced "see'-der") is a division of the Food and Drug Administration (United States), U.S. Food and Drug Administration (FDA) that monitors most drugs as defined in the Food, Drug, and Cosmet ...

(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. Sebelipase alfa is an

orphan drug An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ...

; its effectiveness was published after a phase 3 trial in 2015. The disease of LAL affects < 0.2 in 10,000 people in the EU.

References

{{Portal bar , Medicine Drugs developed by AstraZeneca Biopharmaceuticals Orphan drugs