444:
1571:
31:
1551:
626:
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
625:
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
647:
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
1107:
695:
1100:
174:
1052:
1093:
129:
517:
1616:
559:
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/s1
721:
53:
614:
resulted in "significant improvements in spleen volume, hemoglobin level, liver volume, and platelet count" in untreated adults with
Gaucher disease Type 1.
531:
1655:
606:
826:
1609:
1075:
1034:
991:
1487:
1602:
1635:
551:
669:
775:
1650:
238:
159:
85:
1541:
944:"Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial"
601:
343:
1196:
611:
423:
104:
1582:
1448:
1392:
1382:
1209:
115:
830:
412:
1640:
1493:
1335:
1143:
985:
1085:
1346:
1011:
1407:
1298:
1239:
1235:
586:
439:
1578:
1478:
1283:
258:
122:
1518:
1433:
1276:
1271:
1256:
1231:
1417:
1306:
1226:
1069:
1028:
973:
924:
895:"ELIGLUSTAT TARTRATE: Glucosylceramide Synthase Inhibitor Treatment of Type 1 Gaucher Disease"
875:
392:
332:
211:
198:
186:
43:
1586:
942:
Mistry PK, Lukina E, Ben Turkia H, Amato D, Baris H, Dasouki M, et al. (February 2015).
800:
1387:
1358:
1117:
963:
955:
914:
906:
865:
597:
460:
267:
352:
1645:
1555:
1483:
1397:
1363:
1323:
1293:
1288:
443:
1523:
1412:
1402:
1153:
968:
943:
919:
894:
1629:
1453:
1375:
1163:
726:
292:
77:
910:
1570:
1528:
1513:
1508:
1266:
1251:
1168:
1125:
649:
627:
622:
142:
137:
829:(Press release). U.S. Food and Drug Administration. 19 August 2015. Archived from
670:"Prescription medicines: registration of new chemical entities in Australia, 2015"
63:
1498:
1340:
1311:
1261:
1219:
1173:
749:
71:
1473:
1328:
1316:
1247:
1201:
1191:
1134:
1121:
582:
493:
323:
870:
853:
652:—most often paid for by insurers—in order to remain financially sustainable.
1503:
1468:
1463:
1458:
1443:
1371:
1158:
57:
977:
928:
879:
22:
959:
750:"Cerdelga 84 mg Hard Capsules - Summary of Product Characteristics (SmPC)"
594:
303:
99:
312:
1214:
1148:
590:
278:
30:
1183:
403:
593:, and was approved by the FDA in August 2014. Commonly used as the
372:
1353:
516:
507:
383:
589:. It was discovered at the University of Michigan, developed by
363:
217:
192:
1089:
205:
94:
428:
168:
827:"FDA approves new drug to treat a form of Gaucher disease"
181:
1590:
634:), Genzyme's flagship treatment for Gaucher's disease.
1539:
1054:
New
Genzyme pill will cost patients $ 310,250 a year
600:, the compound is believed to work by inhibition of
1426:
1182:
1133:
505:
492:
459:
454:
422:
402:
382:
362:
342:
322:
302:
277:
257:
229:
158:
153:
128:
114:
84:
70:
52:
42:
37:
854:"Improved inhibitors of glucosylceramide synthase"
291:
1046:
1044:
266:
1005:
1003:
1001:
1610:
1101:
539:CCCCCCCC(=O)N(CN1CCCC1)(C2=CC3=C(C=C2)OCCO3)O
8:
103:
21:
607:Journal of the American Medical Association
1617:
1603:
1108:
1094:
1086:
442:
331:
29:
967:
918:
869:
351:
696:"Product monograph brand safety updates"
1546:
661:
556:
536:
438:
311:
243:
76:
1067:
1026:
983:
674:Therapeutic Goods Administration (TGA)
20:
852:Lee L, Abe A, Shayman JA (May 1999).
411:
391:
62:
7:
1567:
1565:
141:
858:The Journal of Biological Chemistry
371:
282:
1656:Gastrointestinal system drug stubs
1589:. You can help Knowledge (XXG) by
1074:: CS1 maint: overridden setting (
1033:: CS1 maint: overridden setting (
990:: CS1 maint: overridden setting (
14:
1569:
1549:
477:
471:
911:10.1358/dof.2010.035.08.1505566
805:European Medicines Agency (EMA)
564:Key:FJZZPCZKBUKGGU-AUSIDOKSSA-N
1051:Weisman R (2 September 2014),
776:"Cerdelga- eliglustat capsule"
483:
465:
1:
604:. According to an article in
577:, sold under the brand name
1197:phenylalanine ammonia-lyase
612:substrate reduction therapy
1672:
1564:
1010:Garber K (December 2017),
893:Shayman JA (August 2010).
585:used for the treatment of
455:Chemical and physical data
602:glucosylceramide synthase
547:
527:
234:
28:
1636:Beta-Hydroxyamphetamines
1581:article relating to the
871:10.1074/jbc.274.21.14662
1583:gastrointestinal system
1449:Glycerol phenylbutyrate
1393:eladocagene exuparvovec
1383:atidarsagene autotemcel
1210:Carbohydrate metabolism
1651:Transferase inhibitors
1494:Sodium phenylbutyrate
1488:+sodium phenylacetate
1336:iduronate-2-sulfatase
960:10.1001/jama.2015.459
1299:Pegunigalsidase alfa
1240:Cipaglucosidase alfa
1236:Avalglucosidase alfa
1016:, Ann Arbor Observer
1284:alpha-galactosidase
899:Drugs of the Future
833:on 16 February 2017
722:"Genetic disorders"
638:Society and culture
201:(Prescription only)
177:(Prescription only)
25:
1519:Uridine triacetate
1434:Anethole trithione
1277:Velaglucerase alfa
1272:Taliglucerase alfa
1257:glucocerebrosidase
1232:Alglucosidase alfa
1057:, The Boston Globe
1598:
1597:
1537:
1536:
1418:vestronidase alfa
1307:Glycosaminoglycan
1227:alpha-glucosidase
807:. 4 December 2007
587:Gaucher's disease
572:
571:
518:Interactive image
424:CompTox Dashboard
221:
209:
196:
184:
172:
97:
16:Chemical compound
1663:
1619:
1612:
1605:
1573:
1566:
1554:
1553:
1552:
1545:
1388:cerliponase alfa
1359:lysosomal lipase
1347:idursulfase beta
1118:alimentary tract
1110:
1103:
1096:
1087:
1080:
1079:
1073:
1065:
1064:
1062:
1048:
1039:
1038:
1032:
1024:
1023:
1021:
1007:
996:
995:
989:
981:
971:
939:
933:
932:
922:
890:
884:
883:
873:
849:
843:
842:
840:
838:
823:
817:
816:
814:
812:
797:
791:
790:
788:
786:
772:
766:
765:
763:
761:
756:. 8 January 2020
746:
740:
739:
737:
735:
718:
712:
711:
709:
707:
692:
686:
685:
683:
681:
666:
520:
500:
485:
479:
473:
467:
447:
446:
432:
430:
415:
395:
375:
355:
335:
315:
295:
285:
284:
270:
219:
216:
207:
204:
194:
191:
183:
180:
170:
167:
145:
107:
96:
93:
80:
66:
33:
26:
24:
1671:
1670:
1666:
1665:
1664:
1662:
1661:
1660:
1626:
1625:
1624:
1623:
1562:
1560:
1550:
1548:
1540:
1538:
1533:
1484:Sodium benzoate
1422:
1408:pabinafusp alfa
1398:elosulfase alfa
1364:Sebelipase alfa
1324:arylsulfatase B
1294:Agalsidase beta
1289:Agalsidase alfa
1178:
1137:and derivatives
1129:
1114:
1084:
1083:
1066:
1060:
1058:
1050:
1049:
1042:
1025:
1019:
1017:
1009:
1008:
999:
982:
941:
940:
936:
892:
891:
887:
864:(21): 14662–9.
851:
850:
846:
836:
834:
825:
824:
820:
810:
808:
801:"Cerdelga EPAR"
799:
798:
794:
784:
782:
774:
773:
769:
759:
757:
748:
747:
743:
733:
731:
720:
719:
715:
705:
703:
702:. February 2024
694:
693:
689:
679:
677:
668:
667:
663:
658:
645:
640:
620:
568:
565:
560:
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543:
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535:
534:
523:
498:
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476:
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398:
378:
358:
338:
318:
298:
281:
273:
253:
250:
242:
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225:
149:
117:
110:
17:
12:
11:
5:
1669:
1667:
1659:
1658:
1653:
1648:
1643:
1638:
1628:
1627:
1622:
1621:
1614:
1607:
1599:
1596:
1595:
1574:
1559:
1558:
1535:
1534:
1532:
1531:
1526:
1524:Velmanase alfa
1521:
1516:
1511:
1506:
1501:
1496:
1491:
1481:
1476:
1471:
1466:
1461:
1456:
1451:
1446:
1441:
1436:
1430:
1428:
1424:
1423:
1421:
1420:
1415:
1413:pegzilarginase
1410:
1405:
1403:olipudase alfa
1400:
1395:
1390:
1385:
1379:
1378:
1368:
1367:
1350:
1349:
1344:
1332:
1320:
1303:
1302:
1296:
1291:
1280:
1274:
1269:
1264:
1244:
1243:
1223:
1206:
1205:
1188:
1186:
1180:
1179:
1177:
1176:
1171:
1166:
1161:
1156:
1154:Carglumic acid
1151:
1146:
1140:
1138:
1131:
1130:
1115:
1113:
1112:
1105:
1098:
1090:
1082:
1081:
1040:
997:
954:(7): 695–706.
934:
905:(8): 613–620.
885:
844:
818:
792:
767:
741:
713:
687:
676:. 21 June 2022
660:
659:
657:
654:
644:
641:
639:
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619:
616:
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541:
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502:
496:
490:
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480:
474:
468:
463:
457:
456:
452:
451:
449:
448:
440:DTXSID20964175
435:
433:
420:
419:
417:
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406:
400:
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388:
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328:
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275:
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255:
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189:
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134:
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126:
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120:
118:administration
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90:
88:
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81:
74:
68:
67:
60:
50:
49:
46:
40:
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35:
34:
15:
13:
10:
9:
6:
4:
3:
2:
1668:
1657:
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1652:
1649:
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1644:
1642:
1639:
1637:
1634:
1633:
1631:
1620:
1615:
1613:
1608:
1606:
1601:
1600:
1594:
1592:
1588:
1584:
1580:
1575:
1572:
1568:
1563:
1557:
1547:
1543:
1530:
1527:
1525:
1522:
1520:
1517:
1515:
1512:
1510:
1507:
1505:
1502:
1500:
1497:
1495:
1492:
1489:
1485:
1482:
1480:
1477:
1475:
1472:
1470:
1467:
1465:
1462:
1460:
1457:
1455:
1454:Leriglitazone
1452:
1450:
1447:
1445:
1442:
1440:
1437:
1435:
1432:
1431:
1429:
1425:
1419:
1416:
1414:
1411:
1409:
1406:
1404:
1401:
1399:
1396:
1394:
1391:
1389:
1386:
1384:
1381:
1380:
1377:
1376:Asfotase alfa
1373:
1370:
1369:
1365:
1361:
1360:
1355:
1352:
1351:
1348:
1345:
1342:
1338:
1337:
1333:
1330:
1326:
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1321:
1318:
1314:
1313:
1308:
1305:
1304:
1300:
1297:
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1290:
1286:
1285:
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1278:
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1273:
1270:
1268:
1265:
1263:
1259:
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1241:
1237:
1233:
1229:
1228:
1224:
1221:
1217:
1216:
1211:
1208:
1207:
1203:
1199:
1198:
1193:
1190:
1189:
1187:
1185:
1181:
1175:
1172:
1170:
1167:
1165:
1164:Levocarnitine
1162:
1160:
1157:
1155:
1152:
1150:
1147:
1145:
1142:
1141:
1139:
1136:
1132:
1127:
1123:
1119:
1111:
1106:
1104:
1099:
1097:
1092:
1091:
1088:
1077:
1071:
1056:
1055:
1047:
1045:
1041:
1036:
1030:
1015:
1014:
1006:
1004:
1002:
998:
993:
987:
979:
975:
970:
965:
961:
957:
953:
949:
945:
938:
935:
930:
926:
921:
916:
912:
908:
904:
900:
896:
889:
886:
881:
877:
872:
867:
863:
859:
855:
848:
845:
832:
828:
822:
819:
806:
802:
796:
793:
781:
777:
771:
768:
755:
751:
745:
742:
729:
728:
727:Health Canada
723:
717:
714:
701:
700:Health Canada
697:
691:
688:
675:
671:
665:
662:
655:
653:
651:
642:
637:
635:
633:
629:
624:
617:
615:
613:
609:
608:
603:
599:
596:
592:
588:
584:
580:
576:
562:
557:
553:
546:
537:
533:
526:
519:
515:
514:
512:
509:
504:
497:
495:
491:
464:
462:
458:
453:
445:
441:
437:
436:
434:
425:
421:
414:
413:ChEMBL2110588
410:
409:
407:
405:
401:
394:
390:
389:
387:
385:
381:
374:
370:
369:
367:
365:
361:
354:
350:
349:
347:
345:
341:
334:
330:
329:
327:
325:
321:
314:
310:
309:
307:
305:
301:
294:
290:
289:
287:
280:
276:
269:
265:
264:
262:
260:
256:
248:
244:
240:
233:
228:
222: Rx-only
215:
213:
203:
200:
190:
188:
179:
176:
166:
165:
163:
161:
157:
152:
144:
139:
136:
135:
133:
131:
127:
124:
121:
119:
113:
106:
101:
92:
91:
89:
87:
83:
79:
75:
73:
69:
65:
61:
59:
55:
51:
47:
45:
41:
38:Clinical data
36:
32:
27:
19:
1641:Carboxamides
1591:expanding it
1576:
1561:
1529:Zinc acetate
1514:Triheptanoin
1509:Tioctic acid
1438:
1357:
1334:
1322:
1310:
1282:
1267:Imiglucerase
1255:
1252:sphingolipid
1225:
1213:
1195:
1169:Mercaptamine
1144:Ademetionine
1059:, retrieved
1053:
1018:, retrieved
1012:
986:cite journal
951:
947:
937:
902:
898:
888:
861:
857:
847:
835:. Retrieved
831:the original
821:
809:. Retrieved
804:
795:
783:. Retrieved
779:
770:
758:. Retrieved
753:
744:
732:. Retrieved
730:. 9 May 2018
725:
716:
704:. Retrieved
699:
690:
678:. Retrieved
673:
664:
650:orphan drugs
646:
631:
630:(brand name
628:imiglucerase
623:Norman Radin
621:
605:
591:Genzyme Corp
578:
574:
573:
246:
160:Legal status
154:Legal status
86:License data
18:
1499:Teduglutide
1479:Sapropterin
1341:Idursulfase
1312:iduronidase
1262:Alglucerase
1220:Sacrosidase
1192:Amino acids
1174:Metreleptin
1135:Amino acids
1013:Just Reward
501: g·mol
393:CHEBI:82752
268:491833-29-5
249:-octanamide
230:Identifiers
72:MedlinePlus
44:Trade names
1630:Categories
1474:Nitisinone
1439:Eliglustat
1329:Galsulfase
1317:Laronidase
1248:Glycolipid
1202:Pegvaliase
1124:products (
1122:metabolism
1020:17 January
785:30 January
760:30 January
656:References
583:medication
575:Eliglustat
506:3D model (
494:Molar mass
353:DR40J4WA67
324:ChemSpider
259:CAS Number
239:IUPAC name
105:Eliglustat
23:Eliglustat
1504:Trientine
1469:Nedosiran
1464:Miglustat
1459:Lumasiran
1444:Givosiran
1372:Phosphate
1159:Glutamine
811:14 August
643:Economics
610:the oral
116:Routes of
64:Monograph
58:Drugs.com
1556:Medicine
1070:citation
1029:citation
978:25688781
929:22563139
880:10329660
780:DailyMed
734:13 April
706:24 March
680:10 April
632:Cerezyme
595:tartrate
579:Cerdelga
333:28475348
304:DrugBank
293:23652731
130:ATC code
123:By mouth
100:DailyMed
48:Cerdelga
1215:sucrase
1184:Enzymes
1149:Betaine
1061:18 July
969:4962880
920:3340614
837:18 July
618:History
581:, is a
499:404.551
461:Formula
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279:PubChem
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