391:
suggested continued improvement in three participants and stabilization in the others. Two participants in the vestronidase alfa development program experienced marked improvement in pulmonary function. Overall, the results observed would not have been anticipated in the absence of treatment. The effect of vestronidase alfa on the central nervous system manifestations of MPS VII has not been determined.
1248:
691:
628:
398:
The benefit and side effects of vestronidase alfa were based primarily on one trial. Participants were randomly assigned to four groups. Three groups of participants received placebo treatment before starting vestronidase alfa treatment and one group received vestronidase alfa only. vestronidase alfa
390:
mg/kg once every two weeks for up to 164 weeks. Efficacy was primarily assessed via the six-minute walk test in ten participants who could perform the test. After 24 weeks of treatment, the mean difference in distance walked relative to placebo was 18 meters. Additional follow-up for up to 120 weeks
402:
The benefit of 24 weeks of vestronidase alfa treatment was primarily evaluated by the 6-minute walking test (6MWT) and compared to placebo treatment in ten participants who could perform the test. The 6MWT measured the distance a patient could walk on a flat surface in 6 minutes. An additional
385:
The safety and efficacy of vestronidase alfa were established in a clinical trial and expanded access protocols enrolling a total of 23 participants ranging from five months to 25 years of age. Participants received treatment with vestronidase alfa at doses up to
406:
The application for vestronidase alfa was granted fast track designation, orphan drug designation, and a rare pediatric disease priority review voucher. This was the twelfth rare pediatric disease priority review voucher issued.
804:
394:
The FDA approved vestronidase alfa-vjbk based primarily on evidence from one clinical trial (NCT02230566) of 12 participants with mucopolysaccharidosis VII. The trial was conducted at four sites in the United States.
797:
399:
or placebo were given once every two weeks as intravenous (IV) infusions. Neither participants nor healthcare providers knew which treatment was given until after the trial was competed.
790:
613:
123:
63:
776:
for "A Phase 3 Study of UX003 Recombinant Human
Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)" at
491:
Text was copied from this source which is copyright
European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
1184:
605:
343:
type VII (MPS VII), also known as Sly syndrome. MPS VII is an extremely rare, progressive condition that affects most tissues and organs.
676:
339:. It was approved in the United States in November 2017, to treat children and adults with an inherited metabolic condition called
377:
Mepsevii is indicated for the treatment of non-neurological manifestations of
Mucopolysaccharidosis VII (MPS VII; Sly syndrome).
741:
153:
81:
100:
1273:
1238:
717:
670:
609:
411:
362:
218:
893:
418:, and required the manufacturer to conduct a post-marketing study to evaluate the long-term safety of the product.
476:
366:
1145:
1089:
1079:
906:
111:
501:
436:"FDA-sourced list of all drugs with black box warnings (Use Download Full Results and View Query links.)"
1268:
1190:
1032:
840:
340:
782:
1043:
1104:
995:
936:
932:
435:
346:
The most common side effects after treatment with vestronidase alfa include infusion site reactions,
1175:
980:
198:
1215:
1130:
973:
968:
953:
928:
777:
336:
1003:
923:
558:
73:
45:
471:
207:
1084:
1055:
814:
548:
540:
263:
227:
1252:
1180:
1094:
1060:
1020:
990:
985:
664:
1220:
1109:
1099:
850:
553:
528:
17:
1262:
1150:
1072:
860:
695:
632:
168:
1225:
1210:
1205:
963:
948:
865:
822:
332:
136:
131:
772:
33:
1195:
1037:
1008:
958:
916:
870:
355:
544:
1170:
1135:
1025:
1013:
944:
898:
888:
831:
818:
415:
328:
302:
1200:
1165:
1160:
1155:
1140:
1068:
855:
708:
351:
67:
562:
347:
95:
911:
845:
880:
247:
1050:
694:
This article incorporates text from this source, which is in the
631:
This article incorporates text from this source, which is in the
369:. It was approved for use in the European Union in August 2018.
238:
177:
786:
162:
90:
443:
529:"Vestronidase Alfa: A Review in Mucopolysaccharidosis VII"
606:"FDA approves treatment for rare genetic enzyme disorder"
1236:
1123:
879:
830:
403:follow-up using 6MWT was done for up to 120 weeks.
301:
262:
257:
237:
217:
197:
192:
152:
147:
122:
110:
80:
62:
54:
44:
39:
335:. It is a recombinant form of the human enzyme
466:
464:
462:
460:
206:
659:
657:
655:
653:
651:
649:
647:
645:
643:
641:
798:
8:
99:
32:
185:In general: ℞ (Prescription only)
805:
791:
783:
600:
598:
596:
594:
592:
552:
226:
590:
588:
586:
584:
582:
580:
578:
576:
574:
572:
1243:
742:"Mepsevii- vestronidase alfa injection"
427:
527:McCafferty EH, Scott LJ (April 2019).
414:(FDA) granted approval of Mepsevii to
31:
679:from the original on 10 December 2019
616:from the original on 10 December 2019
72:
7:
506:Union Register of medicinal products
358:(sudden, severe allergic reaction).
135:
246:
25:
1246:
689:
626:
280:
274:
709:New Drug Therapy Approvals 2017
665:"Drug Trial Snapshot: Mepsevii"
416:Ultragenyx Pharmaceutical, Inc
292:
286:
268:
1:
718:Food and Drug Administration
671:Food and Drug Administration
610:Food and Drug Administration
412:Food and Drug Administration
363:Food and Drug Administration
894:phenylalanine ammonia-lyase
365:(FDA) considers it to be a
1290:
545:10.1007/s40259-019-00344-7
258:Chemical and physical data
612:(FDA). 15 November 2017.
477:European Medicines Agency
367:first-in-class medication
323:, sold under brand name
1146:Glycerol phenylbutyrate
1090:eladocagene exuparvovec
1080:atidarsagene autotemcel
907:Carbohydrate metabolism
770:Clinical trial number
722:(Report). January 2018
608:(Press release). U.S.
58:Vestronidase alfa-vjbk
18:Vestronidase alfa-vjbk
1191:Sodium phenylbutyrate
1185:+sodium phenylacetate
1033:iduronate-2-sulfatase
341:mucopolysaccharidosis
331:for the treatment of
1274:Recombinant proteins
996:Pegunigalsidase alfa
937:Cipaglucosidase alfa
933:Avalglucosidase alfa
981:alpha-galactosidase
675:. 4 December 2017.
481:. 17 September 2018
36:
27:Pharmaceutical drug
1216:Uridine triacetate
1131:Anethole trithione
974:Velaglucerase alfa
969:Taliglucerase alfa
954:glucocerebrosidase
929:Alglucosidase alfa
778:ClinicalTrials.gov
748:. 19 November 2017
337:beta-glucuronidase
1234:
1233:
1115:vestronidase alfa
1004:Glycosaminoglycan
924:alpha-glucosidase
321:Vestronidase alfa
318:
317:
181:
166:
101:Vestronidase_alfa
93:
34:Vestronidase alfa
16:(Redirected from
1281:
1251:
1250:
1249:
1242:
1085:cerliponase alfa
1056:lysosomal lipase
1044:idursulfase beta
815:alimentary tract
807:
800:
793:
784:
758:
757:
755:
753:
738:
732:
731:
729:
727:
713:
705:
699:
693:
692:
688:
686:
684:
661:
636:
630:
629:
625:
623:
621:
602:
567:
566:
556:
524:
518:
517:
515:
513:
508:. 27 August 2018
498:
492:
490:
488:
486:
468:
455:
454:
452:
450:
440:nctr-crs.fda.gov
432:
389:
313:
311:
294:
288:
282:
276:
270:
250:
230:
210:
179:
176:
171:
164:
161:
139:
103:
92:
89:
76:
37:
35:
21:
1289:
1288:
1284:
1283:
1282:
1280:
1279:
1278:
1259:
1258:
1257:
1247:
1245:
1237:
1235:
1230:
1181:Sodium benzoate
1119:
1105:pabinafusp alfa
1095:elosulfase alfa
1061:Sebelipase alfa
1021:arylsulfatase B
991:Agalsidase beta
986:Agalsidase alfa
875:
834:and derivatives
826:
811:
767:
762:
761:
751:
749:
740:
739:
735:
725:
723:
711:
707:
706:
702:
690:
682:
680:
663:
662:
639:
627:
619:
617:
604:
603:
570:
526:
525:
521:
511:
509:
500:
499:
495:
484:
482:
472:"Mepsevii EPAR"
470:
469:
458:
448:
446:
434:
433:
429:
424:
387:
383:
375:
309:
307:
297:
291:
285:
279:
273:
253:
233:
213:
188:
169:
143:
113:
106:
28:
23:
22:
15:
12:
11:
5:
1287:
1285:
1277:
1276:
1271:
1261:
1260:
1256:
1255:
1232:
1231:
1229:
1228:
1223:
1221:Velmanase alfa
1218:
1213:
1208:
1203:
1198:
1193:
1188:
1178:
1173:
1168:
1163:
1158:
1153:
1148:
1143:
1138:
1133:
1127:
1125:
1121:
1120:
1118:
1117:
1112:
1110:pegzilarginase
1107:
1102:
1100:olipudase alfa
1097:
1092:
1087:
1082:
1076:
1075:
1065:
1064:
1047:
1046:
1041:
1029:
1017:
1000:
999:
993:
988:
977:
971:
966:
961:
941:
940:
920:
903:
902:
885:
883:
877:
876:
874:
873:
868:
863:
858:
853:
851:Carglumic acid
848:
843:
837:
835:
828:
827:
812:
810:
809:
802:
795:
787:
781:
780:
766:
765:External links
763:
760:
759:
733:
700:
637:
568:
539:(2): 233–240.
519:
493:
456:
426:
425:
423:
420:
382:
379:
374:
371:
316:
315:
305:
299:
298:
295:
289:
283:
277:
271:
266:
260:
259:
255:
254:
252:
251:
243:
241:
235:
234:
232:
231:
223:
221:
215:
214:
212:
211:
203:
201:
195:
194:
190:
189:
187:
186:
183:
174:
158:
156:
150:
149:
145:
144:
142:
141:
128:
126:
120:
119:
116:
114:administration
108:
107:
105:
104:
86:
84:
78:
77:
70:
60:
59:
56:
52:
51:
48:
42:
41:
26:
24:
14:
13:
10:
9:
6:
4:
3:
2:
1286:
1275:
1272:
1270:
1267:
1266:
1264:
1254:
1244:
1240:
1227:
1224:
1222:
1219:
1217:
1214:
1212:
1209:
1207:
1204:
1202:
1199:
1197:
1194:
1192:
1189:
1186:
1182:
1179:
1177:
1174:
1172:
1169:
1167:
1164:
1162:
1159:
1157:
1154:
1152:
1151:Leriglitazone
1149:
1147:
1144:
1142:
1139:
1137:
1134:
1132:
1129:
1128:
1126:
1122:
1116:
1113:
1111:
1108:
1106:
1103:
1101:
1098:
1096:
1093:
1091:
1088:
1086:
1083:
1081:
1078:
1077:
1074:
1073:Asfotase alfa
1070:
1067:
1066:
1062:
1058:
1057:
1052:
1049:
1048:
1045:
1042:
1039:
1035:
1034:
1030:
1027:
1023:
1022:
1018:
1015:
1011:
1010:
1005:
1002:
1001:
997:
994:
992:
989:
987:
983:
982:
978:
975:
972:
970:
967:
965:
962:
960:
956:
955:
950:
946:
943:
942:
938:
934:
930:
926:
925:
921:
918:
914:
913:
908:
905:
904:
900:
896:
895:
890:
887:
886:
884:
882:
878:
872:
869:
867:
864:
862:
861:Levocarnitine
859:
857:
854:
852:
849:
847:
844:
842:
839:
838:
836:
833:
829:
824:
820:
816:
808:
803:
801:
796:
794:
789:
788:
785:
779:
775:
774:
769:
768:
764:
747:
743:
737:
734:
721:
719:
710:
704:
701:
697:
696:public domain
678:
674:
672:
666:
660:
658:
656:
654:
652:
650:
648:
646:
644:
642:
638:
634:
633:public domain
615:
611:
607:
601:
599:
597:
595:
593:
591:
589:
587:
585:
583:
581:
579:
577:
575:
573:
569:
564:
560:
555:
550:
546:
542:
538:
534:
530:
523:
520:
507:
503:
502:"Mepsevii PI"
497:
494:
480:
478:
473:
467:
465:
463:
461:
457:
445:
441:
437:
431:
428:
421:
419:
417:
413:
408:
404:
400:
396:
392:
380:
378:
372:
370:
368:
364:
359:
357:
353:
349:
344:
342:
338:
334:
330:
326:
322:
306:
304:
300:
267:
265:
261:
256:
249:
245:
244:
242:
240:
236:
229:
225:
224:
222:
220:
216:
209:
205:
204:
202:
200:
196:
191:
184:
182: Rx-only
175:
172:
160:
159:
157:
155:
151:
146:
138:
133:
130:
129:
127:
125:
121:
117:
115:
109:
102:
97:
88:
87:
85:
83:
79:
75:
71:
69:
65:
61:
57:
53:
49:
47:
43:
40:Clinical data
38:
30:
19:
1269:Orphan drugs
1226:Zinc acetate
1211:Triheptanoin
1206:Tioctic acid
1114:
1054:
1031:
1019:
1007:
979:
964:Imiglucerase
952:
949:sphingolipid
922:
910:
892:
866:Mercaptamine
841:Ademetionine
771:
750:. Retrieved
745:
736:
726:16 September
724:. Retrieved
715:
703:
681:. Retrieved
668:
618:. Retrieved
536:
532:
522:
510:. Retrieved
505:
496:
483:. Retrieved
475:
447:. Retrieved
439:
430:
409:
405:
401:
397:
393:
384:
376:
373:Medical uses
360:
345:
333:Sly syndrome
324:
320:
319:
208:1638194-78-1
154:Legal status
148:Legal status
82:License data
29:
1196:Teduglutide
1176:Sapropterin
1038:Idursulfase
1009:iduronidase
959:Alglucerase
917:Sacrosidase
889:Amino acids
871:Metreleptin
832:Amino acids
773:NCT02230566
485:28 February
356:anaphylaxis
314: g·mol
193:Identifiers
55:Other names
46:Trade names
1263:Categories
1171:Nitisinone
1136:Eliglustat
1026:Galsulfase
1014:Laronidase
945:Glycolipid
899:Pegvaliase
821:products (
819:metabolism
683:9 December
620:9 December
449:22 October
422:References
329:medication
303:Molar mass
228:7XZ4062R17
199:CAS Number
1201:Trientine
1166:Nedosiran
1161:Miglustat
1156:Lumasiran
1141:Givosiran
1069:Phosphate
856:Glutamine
352:urticaria
118:Injection
112:Routes of
74:Monograph
68:Drugs.com
1253:Medicine
752:5 August
746:DailyMed
677:Archived
614:Archived
563:30848434
533:BioDrugs
361:The US.
350:, rash (
348:diarrhea
325:Mepsevii
124:ATC code
96:DailyMed
50:Mepsevii
912:sucrase
881:Enzymes
846:Betaine
554:6469592
512:29 June
410:The US
381:History
327:, is a
264:Formula
173:Rx-only
170:WARNING
140:)
134: (
132:A16AB18
98::
1239:Portal
813:Other
561:
551:
388:
354:) and
248:D11004
167:
94:
1124:Other
1051:Lipid
720:(FDA)
716:U.S.
712:(PDF)
673:(FDA)
669:U.S.
479:(EMA)
817:and
754:2020
728:2020
685:2019
622:2019
559:PMID
514:2024
487:2020
451:2023
278:4996
272:3308
239:KEGG
219:UNII
64:AHFS
823:A16
549:PMC
541:doi
444:FDA
312:.49
310:562
290:940
284:874
137:WHO
1265::
1071::
1053::
1006::
951::
935:,
931:,
909::
891::
744:.
714:.
667:.
640:^
571:^
557:.
547:.
537:33
535:.
531:.
504:.
474:.
459:^
442:.
438:.
308:72
296:16
178:EU
163:US
91:US
1241::
1187:)
1183:(
1063:)
1059:(
1040:)
1036:(
1028:)
1024:(
1016:)
1012:(
998:)
984:(
976:)
957:(
947:/
939:)
927:(
919:)
915:(
901:)
897:(
825:)
806:e
799:t
792:v
756:.
730:.
698:.
687:.
635:.
624:.
565:.
543::
516:.
489:.
453:.
386:4
293:S
287:O
281:N
275:H
269:C
180::
165::
66:/
20:)
Text is available under the Creative Commons Attribution-ShareAlike License. Additional terms may apply.