Eliglustat, sold under the brand name Cerdelga, is 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 ...

used for the treatment of

Gaucher's disease Gaucher's disease or Gaucher disease () (GD) is a genetic disorder A genetic disorder is a health problem caused by one or more abnormalities in the genome. It can be caused by a mutation in a single gene (monogenic) or multiple genes (polyg ...

. It was discovered at the University of Michigan, developed by Genzyme Corp, and was approved by the FDA in August 2014. Commonly used as the

tartrate A tartrate is a salt or ester of the organic compound tartaric acid, a dicarboxylic acid. The formula of the tartrate dianion is O−OC-CH(OH)-CH(OH)-COO− or C4H4O62−. The main forms of tartrates used commercially are pure crystalline ta ...

salt Salt is a mineral composed primarily of sodium chloride (NaCl), a chemical compound belonging to the larger class of salts; salt in the form of a natural crystalline mineral is known as rock salt or halite. Salt is present in vast quantitie ...

, the compound is believed to work by inhibition of

glucosylceramide synthase Ceramide glucosyltransferase (or glucosylceramide synthase) is a glucosyltransferase enzyme involved in the production of glucocerebroside. It is classified under . It is inhibited by miglustat and eliglustat, both drugs developed for the treatm ...

. According to an article in ''

Journal of the American Medical Association ''The Journal of the American Medical Association'' (''JAMA'') is a peer-reviewed medical journal published 48 times a year by the American Medical Association. It publishes original research, reviews, and editorials covering all aspects of bio ...

'' the oral

substrate reduction therapy Substrate reduction therapy offers an approach to treatment of certain metabolic disorders, especially glycogen storage diseases and lysosomal storage disorders. In a storage disorder, a critical failure in a metabolic pathway prevents cellular br ...

resulted in "significant improvements in spleen volume, hemoglobin level, liver volume, and platelet count" in untreated adults with Gaucher disease Type 1.

History

Norman Radin Norman S. Radin was a neurochemist who, along with Jim Shayman, developed eliglustat, a drug for treating Gaucher's disease. Born in New York City, he received a B.S. in 1941 and Ph.D. in 1949 from Columbia University, later becoming an associate ...

began exploring the possibility of inhibiting the synthesis of lipid substrates involved in Gaucher's disease as early as 1982, and, in collaboration with the laboratory of Jim Shayman, found several candidate inhibitors in the mid-1990s. Genzyme initially rejected the candidates developed by Radin and Shayman, but after a news broke of a competitor developing a new treatment for Gaucher's disease, licensed the Radin/Shayman patents in 2000. Eliglustat did not receive FDA approval for another 14 years, a delay that Shayman speculated was due to some company leaders not being fully committed to developing a drug that would compete with

imiglucerase Imiglucerase is a medication used in the treatment of Gaucher's disease. It is a recombinant DNA-produced analogue of the human enzyme β-glucocerebrosidase. Cerezyme is a freeze-dried medicine containing imiglucerase, manufactured by Genzyme C ...

(brand name ''Cerezyme''), Genzyme's flagship treatment for Gaucher's disease.

Society and culture

Economics

In 2014, the annual cost of eliglustat taken orally twice a day was $310,250. Cerezyme cost about $300,000 for the intravenous medication if taken twice a month. Manufacturing costs for eliglustat are slightly lower than for imiglucerase. Genzyme maintains higher prices for

orphan drugs 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 ...

—most often paid for by insurers—in order to remain financially sustainable.

References

External links

* Carboxamides Phenylethanolamines Sanofi Transferase inhibitors {{gastrointestinal-drug-stub