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

enzyme Enzymes () are proteins that act as biological catalysts by accelerating chemical reactions. The molecules upon which enzymes may act are called substrates, and the enzyme converts the substrates into different molecules known as products. A ...

lysosomal acid lipase (LAL) that is used as a

medication A medication (also called medicament, medicine, pharmaceutical drug, medicinal drug or simply drug) is a drug used to diagnose, cure, treat, or prevent disease. Drug therapy (pharmacotherapy) is an important part of the medical field and re ...

for the treatment of

lysosomal acid lipase deficiency Lysosomal acid lipase deficiency (LAL deficiency or LAL-D) is an autosomal recessive inborn error of metabolism that results in the body not producing enough active lysosomal acid lipase (LAL) enzyme. This enzyme plays an important role in breakin ...

(LAL-D). Text was copied from this source which is © 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 Alexion Pharmaceuticals is an American pharmaceutical company headquartered in Boston, Massachusetts that specializes in orphan drugs to treat rare diseases. It became an independent subsidiary of AstraZeneca in 2021. Its products include eculi ...

in 2015. For its production,

chicken The chicken (''Gallus gallus domesticus'') is a domesticated junglefowl species, with attributes of wild species such as the grey and the Ceylon junglefowl that are originally from Southeastern Asia. Rooster or cock is a term for an adult m ...

s are

genetically modified Genetic engineering, also called genetic modification or genetic manipulation, is the modification and manipulation of an organism's genes using technology. It is a set of Genetic engineering techniques, technologies used to change the gene ...

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 arou ...

. After extraction and purification it becomes available as the medication. On 8 December 2015 the

FDA The United States Food and Drug Administration (FDA or US FDA) is a federal agency of the Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food s ...

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 U.S. Food and Drug Administration (FDA) that monitors most drugs as defined in the Food, Drug, and Cosmetic Act. Some biological products are also leg ...

(CDER) approved the human therapeutic application of the medication, while the

Center for Veterinary Medicine The Center for Veterinary Medicine (CVM) is a branch of the U.S. Food and Drug Administration (FDA) that regulates the manufacture and distribution of food, food additives, and drugs that will be given to animals. These include animals from which ...

(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 pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment of ...

; 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

External links

* AstraZeneca brands Biopharmaceuticals Breakthrough therapy Orphan drugs {{gastrointestinal-drug-stub