1342:
898:
449:
In 2014 the U.S. Food and Drug
Administration announced the approval of alglucosidase alfa for treatment of people with infantile-onset Pompe disease, including people who are less than eight years of age. In addition, the
891:
169:
499:
Some
American health plans have refused to subsidize alglucosidase alfa for adults because it lacks approval for treatment in adults, as well as its high cost (US$ 300,000 per year for life).
884:
124:
384:
532:
62:
737:
593:
767:
807:
856:
655:
451:
1278:
623:
559:
417:
524:
834:
209:
154:
80:
99:
808:"FDA expands approval of drug to treat Pompe disease to patients of all ages; removes risk mitigation strategy requirements"
1332:
282:
1367:
987:
729:
651:
424:
409:
585:
690:"Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease"
502:
In 2015, alglucosidase alfa was ranked the costliest drug per patient, with an average charge of US$ 630,159.
784:
438:
It was approved for medical use in the United States in April 2006, as
Myozyme and in May 2010, as Lumizyme.
1239:
1183:
1173:
1000:
759:
110:
811:
1362:
1284:
1126:
934:
863:
663:
876:
1137:
1198:
1089:
1030:
1026:
31:
1269:
1074:
226:
1309:
1224:
1067:
1062:
1047:
428:
486:. Myozyme boxes carry warnings regarding the possibility of life-threatening allergic response.
1208:
1097:
1017:
711:
586:"Myozyme (alglucosidase alfa) Injectable for intravenous infusion Initial U.S. Approval: 2006"
432:
182:
52:
1178:
1149:
908:
701:
615:
327:
235:
291:
1372:
1346:
1274:
1188:
1154:
1114:
1084:
1079:
72:
688:
Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL, et al. (January 2007).
563:
1314:
1203:
1193:
944:
706:
689:
1356:
1244:
1166:
954:
785:"Center for Drug Evaluation and Research: Application Number: 125291: Summary Review"
475:
826:
1319:
1304:
1299:
1057:
1042:
959:
916:
479:
421:
137:
132:
40:
1289:
1131:
1102:
1052:
1010:
964:
446:
Alglucosidase alfa is indicated for people with Pompe disease (GAA deficiency).
413:
117:
1264:
1229:
1119:
1107:
1038:
992:
982:
925:
912:
366:
266:
1294:
1259:
1254:
1249:
1234:
1162:
949:
467:
463:
66:
715:
246:
94:
255:
1005:
939:
483:
431:
of the enzyme that is deficient in patients affected by Pompe disease,
17:
462:
Common observed adverse reactions to alglucosidase alfa treatment are
974:
311:
1144:
760:"Drug Approval Package: Lumizyme (alglucosidase alfa) NDA #125291"
471:
730:"Drug Approval Package: Myozyme (Alglucosidase Alfa) NDA #125141"
302:
188:
880:
176:
89:
660:
525:"Lumizyme- alglucosidase alfa injection, powder, for solution"
163:
435:. It is the first drug available to treat this disease.
391:
857:"Magellan Rx Management Medical Pharmacy Trend Report"
1330:
519:
517:
515:
1217:
973:
924:
365:
326:
321:
301:
281:
265:
245:
225:
200:
153:
148:
123:
109:
79:
61:
51:
46:
560:"FDA Approves First Treatment for Pompe Disease"
234:
892:
8:
827:"As Costs Rise, New Medicines Face Pushback"
98:
39:
899:
885:
877:
474:. More serious reactions reported include
705:
554:
552:
550:
290:
1337:
511:
452:Risk Evaluation and Mitigation Strategy
254:
214:
645:
643:
641:
38:
810:. FDA. 14 August 2014. Archived from
770:from the original on 30 November 2020
626:from the original on 28 December 2021
596:from the original on 18 December 2022
71:
7:
862:(7th ed.). 2016. Archived from
562:. FDA. 28 April 2006. Archived from
136:
310:
837:from the original on 21 April 2019
740:from the original on 12 April 2021
707:10.1212/01.wnl.0000251268.41188.04
535:from the original on 25 March 2021
427:(LSD). Chemically, the drug is an
25:
764:U.S. Food and Drug Administration
734:U.S. Food and Drug Administration
1340:
418:Glycogen storage disease type II
416:for treatment of Pompe disease (
344:
338:
27:Enzyme replacement therapy drug
650:American Medical Association (
356:
350:
332:
1:
825:Anand G (18 September 2007).
466:, respiratory complications,
792:Food and Drug Administration
454:(REMS) is being eliminated.
404:, sold under the brand name
217:Human glucosidase, prepro-α-
988:phenylalanine ammonia-lyase
1389:
425:lysosomal storage disorder
410:enzyme replacement therapy
322:Chemical and physical data
29:
620:European Medicines Agency
381:
205:
57:Myozyme, Lumizyme, others
30:Not to be confused with
1240:Glycerol phenylbutyrate
1184:eladocagene exuparvovec
1174:atidarsagene autotemcel
1001:Carbohydrate metabolism
831:The Wall Street Journal
1285:Sodium phenylbutyrate
1279:+sodium phenylacetate
1127:iduronate-2-sulfatase
1090:Pegunigalsidase alfa
1031:Cipaglucosidase alfa
1027:Avalglucosidase alfa
656:"Alglucosidase alfa"
622:. 14 February 2001.
531:. 22 February 2020.
408:among others, is an
32:Avalglucosidase alfa
1075:alpha-galactosidase
669:on 12 February 2012
490:Society and culture
172:(Prescription only)
43:
1368:Medical treatments
1310:Uridine triacetate
1225:Anethole trithione
1068:Velaglucerase alfa
1063:Taliglucerase alfa
1048:glucocerebrosidase
1023:Alglucosidase alfa
402:Alglucosidase alfa
100:Alglucosidase alfa
41:Alglucosidase alfa
1328:
1327:
1209:vestronidase alfa
1098:Glycosaminoglycan
1018:alpha-glucosidase
869:on 29 March 2019.
814:on 3 August 2014.
433:alpha-glucosidase
399:
398:
192:
180:
167:
92:
16:(Redirected from
1380:
1345:
1344:
1343:
1336:
1179:cerliponase alfa
1150:lysosomal lipase
1138:idursulfase beta
909:alimentary tract
901:
894:
887:
878:
871:
870:
868:
861:
853:
847:
846:
844:
842:
822:
816:
815:
804:
798:
795:
789:
782:Lay summary in:
779:
777:
775:
756:
750:
749:
747:
745:
726:
720:
719:
709:
685:
679:
678:
676:
674:
668:
667:(Microsoft Word)
662:. Archived from
647:
636:
635:
633:
631:
612:
606:
605:
603:
601:
582:
576:
575:
573:
571:
556:
545:
544:
542:
540:
521:
395:
394:
387:
376:
375:
358:
352:
346:
340:
334:
314:
294:
258:
238:
190:
187:
178:
175:
165:
162:
140:
102:
91:
88:
75:
44:
42:
21:
1388:
1387:
1383:
1382:
1381:
1379:
1378:
1377:
1353:
1352:
1351:
1341:
1339:
1331:
1329:
1324:
1275:Sodium benzoate
1213:
1199:pabinafusp alfa
1189:elosulfase alfa
1155:Sebelipase alfa
1115:arylsulfatase B
1085:Agalsidase beta
1080:Agalsidase alfa
969:
928:and derivatives
920:
905:
875:
874:
866:
859:
855:
854:
850:
840:
838:
824:
823:
819:
806:
805:
801:
787:
783:
773:
771:
758:
757:
753:
743:
741:
728:
727:
723:
687:
686:
682:
672:
670:
666:
649:
648:
639:
629:
627:
614:
613:
609:
599:
597:
584:
583:
579:
569:
567:
558:
557:
548:
538:
536:
523:
522:
513:
508:
497:
492:
460:
444:
390:
388:
385:(what is this?)
382:
373:
371:
361:
355:
349:
343:
337:
317:
297:
277:
261:
241:
221:
218:
213:
212:
196:
144:
112:
105:
35:
28:
23:
22:
15:
12:
11:
5:
1386:
1384:
1376:
1375:
1370:
1365:
1355:
1354:
1350:
1349:
1326:
1325:
1323:
1322:
1317:
1315:Velmanase alfa
1312:
1307:
1302:
1297:
1292:
1287:
1282:
1272:
1267:
1262:
1257:
1252:
1247:
1242:
1237:
1232:
1227:
1221:
1219:
1215:
1214:
1212:
1211:
1206:
1204:pegzilarginase
1201:
1196:
1194:olipudase alfa
1191:
1186:
1181:
1176:
1170:
1169:
1159:
1158:
1141:
1140:
1135:
1123:
1111:
1094:
1093:
1087:
1082:
1071:
1065:
1060:
1055:
1035:
1034:
1014:
997:
996:
979:
977:
971:
970:
968:
967:
962:
957:
952:
947:
945:Carglumic acid
942:
937:
931:
929:
922:
921:
906:
904:
903:
896:
889:
881:
873:
872:
848:
817:
799:
797:
796:
751:
721:
680:
637:
616:"Myozyme EPAR"
607:
577:
566:on 19 May 2006
546:
510:
509:
507:
504:
496:
493:
491:
488:
484:allergic shock
459:
456:
443:
440:
397:
396:
379:
378:
369:
363:
362:
359:
353:
347:
341:
335:
330:
324:
323:
319:
318:
316:
315:
307:
305:
299:
298:
296:
295:
287:
285:
279:
278:
276:
275:
271:
269:
263:
262:
260:
259:
251:
249:
243:
242:
240:
239:
231:
229:
223:
222:
220:
219:
216:
208:
207:
206:
203:
202:
198:
197:
195:
194:
185:
173:
159:
157:
151:
150:
146:
145:
143:
142:
129:
127:
121:
120:
115:
113:administration
107:
106:
104:
103:
85:
83:
77:
76:
69:
59:
58:
55:
49:
48:
26:
24:
14:
13:
10:
9:
6:
4:
3:
2:
1385:
1374:
1371:
1369:
1366:
1364:
1361:
1360:
1358:
1348:
1338:
1334:
1321:
1318:
1316:
1313:
1311:
1308:
1306:
1303:
1301:
1298:
1296:
1293:
1291:
1288:
1286:
1283:
1280:
1276:
1273:
1271:
1268:
1266:
1263:
1261:
1258:
1256:
1253:
1251:
1248:
1246:
1245:Leriglitazone
1243:
1241:
1238:
1236:
1233:
1231:
1228:
1226:
1223:
1222:
1220:
1216:
1210:
1207:
1205:
1202:
1200:
1197:
1195:
1192:
1190:
1187:
1185:
1182:
1180:
1177:
1175:
1172:
1171:
1168:
1167:Asfotase alfa
1164:
1161:
1160:
1156:
1152:
1151:
1146:
1143:
1142:
1139:
1136:
1133:
1129:
1128:
1124:
1121:
1117:
1116:
1112:
1109:
1105:
1104:
1099:
1096:
1095:
1091:
1088:
1086:
1083:
1081:
1077:
1076:
1072:
1069:
1066:
1064:
1061:
1059:
1056:
1054:
1050:
1049:
1044:
1040:
1037:
1036:
1032:
1028:
1024:
1020:
1019:
1015:
1012:
1008:
1007:
1002:
999:
998:
994:
990:
989:
984:
981:
980:
978:
976:
972:
966:
963:
961:
958:
956:
955:Levocarnitine
953:
951:
948:
946:
943:
941:
938:
936:
933:
932:
930:
927:
923:
918:
914:
910:
902:
897:
895:
890:
888:
883:
882:
879:
865:
858:
852:
849:
836:
832:
828:
821:
818:
813:
809:
803:
800:
793:
786:
781:
780:
769:
765:
761:
755:
752:
739:
735:
731:
725:
722:
717:
713:
708:
703:
700:(2): 99–109.
699:
695:
691:
684:
681:
665:
661:
657:
653:
646:
644:
642:
638:
625:
621:
617:
611:
608:
595:
591:
587:
581:
578:
565:
561:
555:
553:
551:
547:
534:
530:
526:
520:
518:
516:
512:
505:
503:
500:
494:
489:
487:
485:
481:
477:
473:
469:
465:
457:
455:
453:
447:
441:
439:
436:
434:
430:
426:
423:
419:
415:
411:
407:
403:
393:
386:
380:
370:
368:
364:
331:
329:
325:
320:
313:
309:
308:
306:
304:
300:
293:
289:
288:
286:
284:
280:
273:
272:
270:
268:
264:
257:
253:
252:
250:
248:
244:
237:
233:
232:
230:
228:
224:
215:
211:
204:
199:
193: Rx-only
186:
184:
174:
171:
161:
160:
158:
156:
152:
147:
139:
134:
131:
130:
128:
126:
122:
119:
116:
114:
108:
101:
96:
87:
86:
84:
82:
78:
74:
70:
68:
64:
60:
56:
54:
50:
47:Clinical data
45:
37:
33:
19:
1363:Orphan drugs
1320:Zinc acetate
1305:Triheptanoin
1300:Tioctic acid
1148:
1125:
1113:
1101:
1073:
1058:Imiglucerase
1046:
1043:sphingolipid
1022:
1016:
1004:
986:
960:Mercaptamine
935:Ademetionine
864:the original
851:
839:. Retrieved
830:
820:
812:the original
802:
791:
772:. Retrieved
763:
754:
742:. Retrieved
733:
724:
697:
693:
683:
671:. Retrieved
664:the original
659:
628:. Retrieved
619:
610:
598:. Retrieved
589:
580:
568:. Retrieved
564:the original
537:. Retrieved
528:
501:
498:
480:lung failure
461:
458:Side effects
448:
445:
442:Medical uses
437:
405:
401:
400:
389:
383:
155:Legal status
149:Legal status
81:License data
36:
1290:Teduglutide
1270:Sapropterin
1132:Idursulfase
1103:iduronidase
1053:Alglucerase
1011:Sacrosidase
983:Amino acids
965:Metreleptin
926:Amino acids
673:18 December
600:18 December
414:orphan drug
377: g·mol
236:420784-05-0
201:Identifiers
118:Intravenous
53:Trade names
1357:Categories
1265:Nitisinone
1230:Eliglustat
1120:Galsulfase
1108:Laronidase
1039:Glycolipid
993:Pegvaliase
915:products (
913:metabolism
506:References
468:infections
367:Molar mass
292:DTI67O9503
267:ChemSpider
227:CAS Number
210:IUPAC name
1295:Trientine
1260:Nedosiran
1255:Miglustat
1250:Lumasiran
1235:Givosiran
1163:Phosphate
950:Glutamine
774:14 August
744:14 August
694:Neurology
539:14 August
495:Economics
464:pneumonia
111:Routes of
73:Monograph
67:Drugs.com
1347:Medicine
835:Archived
768:Archived
738:Archived
716:17151339
624:Archived
594:Archived
590:DailyMed
533:Archived
529:DailyMed
392:(verify)
247:DrugBank
125:ATC code
95:DailyMed
1006:sucrase
975:Enzymes
940:Betaine
406:Myozyme
328:Formula
256:DB01272
141:)
135: (
133:A16AB07
97::
18:Myozyme
1373:Sanofi
1333:Portal
907:Other
841:7 July
714:
630:15 May
570:7 July
429:analog
412:(ERT)
312:D03207
183:℞-only
181:
168:
93:
1218:Other
1145:Lipid
867:(PDF)
860:(PDF)
788:(PDF)
476:heart
472:fever
420:), a
911:and
843:2008
776:2020
746:2020
712:PMID
675:2007
652:USAN
632:2024
602:2022
572:2008
541:2020
482:and
478:and
470:and
422:rare
354:1369
348:1274
342:7262
336:4758
303:KEGG
283:UNII
274:none
63:AHFS
917:A16
702:doi
654:).
374:338
372:105
138:WHO
1359::
1165::
1147::
1100::
1045::
1029:,
1025:,
1003::
985::
833:.
829:.
790:.
766:.
762:.
736:.
732:.
710:.
698:68
696:.
692:.
658:.
640:^
618:.
592:.
588:.
549:^
527:.
514:^
360:35
189:EU
177:US
170:S4
164:AU
90:US
1335::
1281:)
1277:(
1157:)
1153:(
1134:)
1130:(
1122:)
1118:(
1110:)
1106:(
1092:)
1078:(
1070:)
1051:(
1041:/
1033:)
1021:(
1013:)
1009:(
995:)
991:(
919:)
900:e
893:t
886:v
845:.
794:.
778:.
748:.
718:.
704::
677:.
634:.
604:.
574:.
543:.
357:S
351:O
345:N
339:H
333:C
191::
179::
166::
65:/
34:.
20:)
Text is available under the Creative Commons Attribution-ShareAlike License. Additional terms may apply.