Idursulfase (brand name Elaprase), manufactured by
, is a drug used to treat
Hunter syndrome
Hunter syndrome, or mucopolysaccharidosis type II (MPS II), is a rare genetic disorder in which large sugar molecules called glycosaminoglycans (or GAGs or mucopolysaccharides) build up in body tissues. It is a form of lysosomal storage disease. ...
(also called
MPS-II).
It is a purified form of the
lysosomal
A lysosome () is a membrane-bound organelle found in many animal cells. They are spherical vesicles that contain hydrolytic enzymes that can break down many kinds of biomolecules. A lysosome has a specific composition, of both its membrane pr ...
enzyme
Enzymes () are proteins that act as biological catalysts by accelerating chemical reactions. The molecules upon which enzymes may act are called substrate (chemistry), substrates, and the enzyme converts the substrates into different molecule ...
iduronate-2-sulfatase and is produced by
recombinant DNA technology
Molecular cloning is a set of experimental methods in molecular biology that are used to assemble recombinant DNA molecules and to direct their replication within host organisms. The use of the word ''cloning'' refers to the fact that the metho ...
in a human cell line.
It is one of the most expensive drugs ever produced, costing US$567,412 per patient per year.
Barbara Kollmeyer, Marketwatch, Fed. 3, 2016
References
External links
*
Orphan drugs
{{gastrointestinal-drug-stub