Norman Radin 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.[8] 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.[8] 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 (brand name Cerezyme), Genzyme's flagship treatment for Gaucher's disease.[8]
Society and cultureedit
Economicsedit
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.[9] Manufacturing costs for eliglustat are slightly lower than for imiglucerase. Genzyme maintains higher prices for orphan drugs—most often paid for by insurers—in order to remain financially sustainable.[9]
Referencesedit
^"Prescription medicines: registration of new chemical entities in Australia, 2015". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 10 April 2023.
^"Cerdelga 84 mg Hard Capsules - Summary of Product Characteristics (SmPC)". (emc). 8 January 2020. Retrieved 30 January 2021.
^"Cerdelga- eliglustat capsule". DailyMed. Retrieved 30 January 2021.
^ (Press release). U.S. Food and Drug Administration. 19 August 2015. Archived from the original on 16 February 2017. Retrieved 18 July 2015.
^Lee L, Abe A, Shayman JA (May 1999). "Improved inhibitors of glucosylceramide synthase". The Journal of Biological Chemistry. 274 (21): 14662–9. doi:10.1074/jbc.274.21.14662. PMID 10329660.
^Shayman JA (August 2010). "ELIGLUSTAT TARTRATE: Glucosylceramide Synthase Inhibitor Treatment of Type 1 Gaucher Disease". Drugs of the Future. 35 (8): 613–620. doi:10.1358/dof.2010.035.08.1505566. PMC3340614. PMID 22563139.
^Mistry PK, Lukina E, Ben Turkia H, Amato D, Baris H, Dasouki M, et al. (February 2015). "Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial". JAMA. 313 (7): 695–706. doi:10.1001/jama.2015.459. PMC4962880. PMID 25688781.
^ abcGarber K (December 2017), Just Reward, Ann Arbor Observer, retrieved 17 January 2021
^ abWeisman R (2 September 2014), New Genzyme pill will cost patients $310,250 a year, The Boston Globe, retrieved 18 July 2015
External linksedit
"Eliglustat". Drug Information Portal. U.S. National Library of Medicine.
eliglustat, sold, under, brand, name, cerdelga, medication, used, treatment, gaucher, disease, discovered, university, michigan, developed, genzyme, corp, approved, august, 2014, commonly, used, tartrate, salt, compound, believed, work, inhibition, glucosylcer. Eliglustat sold under the brand name Cerdelga is a medication used for the treatment of Gaucher s disease It was discovered at the University of Michigan developed by Genzyme Corp and was approved by the FDA in August 2014 4 Commonly used as the tartrate salt the compound is believed to work by inhibition of glucosylceramide synthase 5 6 According to an article in Journal of the American Medical Association the oral substrate reduction therapy resulted in significant improvements in spleen volume hemoglobin level liver volume and platelet count in untreated adults with Gaucher disease Type 1 7 EliglustatClinical dataTrade namesCerdelgaAHFS Drugs comMonographMedlinePlusa618038License dataEU EMA by INN US DailyMed EliglustatRoutes ofadministrationBy mouthATC codeA16AX10 WHO Legal statusLegal statusAU S4 Prescription only 1 UK POM Prescription only 2 US only 3 EU Rx onlyIdentifiersIUPAC name N 1R 2R 1 2 3 Dihydro 1 4 benzodioxin 6 yl 1 hydroxy 3 1 pyrrolidinyl 2 propanyl octanamideCAS Number491833 29 5PubChem CID23652731DrugBankDB09039ChemSpider28475348UNIIDR40J4WA67KEGGD09893ChEBICHEBI 82752 YChEMBLChEMBL2110588CompTox Dashboard EPA DTXSID20964175Chemical and physical dataFormulaC 23H 36N 2O 4Molar mass404 551 g mol 13D model JSmol Interactive imageSMILES CCCCCCCC O N C H CN1CCCC1 C H C2 CC3 C C C2 OCCO3 OInChI InChI 1S C23H36N2O4 c1 2 3 4 5 6 9 22 26 24 19 17 25 12 7 8 13 25 23 27 18 10 11 20 21 16 18 29 15 14 28 20 h10 11 16 19 23 27H 2 9 12 15 17H2 1H3 H 24 26 t19 23 m1 s1Key FJZZPCZKBUKGGU AUSIDOKSSA N Contents 1 History 2 Society and culture 2 1 Economics 3 References 4 External linksHistory editNorman Radin 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 8 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 8 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 brand name Cerezyme Genzyme s flagship treatment for Gaucher s disease 8 Society and culture editEconomics edit 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 9 Manufacturing costs for eliglustat are slightly lower than for imiglucerase Genzyme maintains higher prices for orphan drugs most often paid for by insurers in order to remain financially sustainable 9 References edit Prescription medicines registration of new chemical entities in Australia 2015 Therapeutic Goods Administration TGA 21 June 2022 Retrieved 10 April 2023 Cerdelga 84 mg Hard Capsules Summary of Product Characteristics SmPC emc 8 January 2020 Retrieved 30 January 2021 Cerdelga eliglustat capsule DailyMed Retrieved 30 January 2021 FDA approves new drug to treat a form of Gaucher disease Press release U S Food and Drug Administration 19 August 2015 Archived from the original on 16 February 2017 Retrieved 18 July 2015 Lee L Abe A Shayman JA May 1999 Improved inhibitors of glucosylceramide synthase The Journal of Biological Chemistry 274 21 14662 9 doi 10 1074 jbc 274 21 14662 PMID 10329660 Shayman JA August 2010 ELIGLUSTAT TARTRATE Glucosylceramide Synthase Inhibitor Treatment of Type 1 Gaucher Disease Drugs of the Future 35 8 613 620 doi 10 1358 dof 2010 035 08 1505566 PMC 3340614 PMID 22563139 Mistry PK Lukina E Ben Turkia H Amato D Baris H Dasouki M et al February 2015 Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1 the ENGAGE randomized clinical trial JAMA 313 7 695 706 doi 10 1001 jama 2015 459 PMC 4962880 PMID 25688781 a b c Garber K December 2017 Just Reward Ann Arbor Observer retrieved 17 January 2021 a b Weisman R 2 September 2014 New Genzyme pill will cost patients 310 250 a year The Boston Globe retrieved 18 July 2015External links edit Eliglustat Drug Information Portal U S National Library of Medicine Portal nbsp Medicine nbsp This drug article relating to the gastrointestinal system is a stub You can help Wikipedia by expanding it vte Retrieved from https en wikipedia org w index php title Eliglustat amp oldid 1186346894, wikipedia, wiki, book, books, library,
