417:(EMA) adopted a positive opinion, recommending the granting of a conditional marketing authorization for the medicinal product Casgevy, intended for the treatment of transfusion‑dependent β‑thalassemia and sickle cell disease. As Casgevy is an advanced therapy medicinal product, the CHMP positive opinion is based on an assessment by the Committee for Advanced Therapies. The applicant for this medicinal product is Vertex Pharmaceuticals (Ireland) Limited. Exagamglogene autotemcel was approved for medical use in the European Union in February 2024.
373:
prior to screening. The primary efficacy outcome was freedom from severe vaso-occlusive crisis episodes for at least twelve consecutive months during the 24-month follow-up period. A total of 44 participants were treated with exagamglogene autotemcel. Of the 31 participants with sufficient follow-up time to be evaluable, 29 (93.5%) achieved this outcome. All treated participants achieved successful engraftment with no participants experiencing graft failure or
355:
myeloablative conditioning (high-dose chemotherapy), a process that removes cells from the bone marrow so they can be replaced with the modified cells in exagamglogene autotemcel. The modified blood stem cells are transplanted back into the recipient where they engraft (attach and multiply) within the bone marrow and increase the production of fetal hemoglobin (HbF), a type of hemoglobin that facilitates oxygen delivery.
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The gene therapy is made from the recipient's own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant. Prior to treatment, the recipient's own stem cells are collected, and then the recipient must undergo
372:
The safety and effectiveness of exagamglogene autotemcel were evaluated in an ongoing single-arm, multi-center trial in adult and adolescent participants with sickle cell disease. Participants had a history of at least two protocol-defined severe vaso-occlusive crises during each of the two years
316:
The treatment was approved in the United
Kingdom for the treatment of sickle cell disease and transfusion-dependent beta thalassemia in November 2023. It was approved in the United States for the treatment of sickle cell disease in December 2023 and for the treatment of transfusion-dependent beta
907:
363:
The most common side effects observed in clinical studies included low levels of platelets and white blood cells, mouth sores, nausea, musculoskeletal pain, abdominal pain, vomiting, febrile neutropenia (fever and low white blood cell count), headache and itching.
878:"Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, Casgevy (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia"
312:
The most common side effects include low levels of platelets and white blood cells, mouth sores, nausea, musculoskeletal pain, abdominal pain, vomiting, febrile neutropenia (fever and low white blood cell count), headache, and itching.
903:
796:
690:
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429:. The cost effectiveness threshold of the therapy in the US is estimated to be between $ 1.35 million and $ 2.05 million depending on perspective (healthcare vs limited societal) and assuming the
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for the treatment of transfusion-dependent beta thalassemia and sickle cell disease in patients aged 12 years and older who should be treated with
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Text was copied from this source which is copyright
European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
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In the US, exagamglogene autotemcel is indicated for the treatment of sickle cell disease in people aged 12 years and older with recurrent
1118:
1299:
904:"Vertex and CRISPR Therapeutics Announce US FDA Approval of Casgevy (exagamglogene autotemcel) for the Treatment of Sickle Cell Disease"
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396:, and regenerative medicine advanced therapy designations. The FDA granted approval of Casgevy to Vertex Pharmaceuticals.
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786:"MHRA authorises world-first gene therapy that aims to cure sickle-cell disease and transfusion-dependent β-thalassemia"
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1239:"Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia"
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1204:"Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Severe Sickle Cell Disease"
1202:
Frangoul H, Locatelli F, Bhatia M, Mapara MY, Molinari L, Sharma A, et al. (November 2022).
1024:"In historic decision, FDA approves a CRISPR-based medicine for treatment of sickle cell disease"
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430:
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Locatelli F, Lang P, Li A, Corbacioglu S, de la Fuente J, Wall DA, et al. (November 2022).
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683:"FDA advisers see no roadblocks for gene-editing treatment for sickle cell disease"
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Exagamglogene autotemcel is the first cell-based gene therapy treatment utilizing
1428:
1418:
1054:"ICER Publishes Final Evidence Report on Gene Therapies for Sickle Cell Disease"
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742:"FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease"
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1150:"UK first to approve CRISPR treatment for diseases: what you need to know"
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1119:"Life-changing blood disease treatment approved at £1m cost per patient"
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820:"The world's first CRISPR therapy is approved: who will receive it?"
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This article incorporates text from this source, which is in the
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This article incorporates text from this source, which is in the
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880:(Press release). Vertex Pharmaceuticals. 16 November 2023.
384:(FDA) granted the application for exagamglogene autotemcel
906:(Press release). Vertex Pharmaceuticals. 8 December 2023.
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but for whom a suitable stem cell donor is not available.
535:"Casgevy- exagamglogene autotemcel injection, suspension"
502:"Casgevy 4–13 x 10Exp6 cells/mL dispersion for infusion"
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Medicines and
Healthcare products Regulatory Agency
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Medicines and
Healthcare products Regulatory Agency
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1117:Pinkstone J, Searles M (16 November 2023).
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1091:Institute for Clinical and Economic Review
1059:Institute for Clinical and Economic Review
1281:"Exagamglogene Autotemcel (Code C169042)"
1254:
1219:
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475:(MHRA). 15 November 2023. Archived from
1516:
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342:hematopoietic stem cell transplantation
336:In the UK, exagamglogene autotemcel is
229:
86:
31:
1129:from the original on 10 December 2023
1098:from the original on 14 November 2023
1003:from the original on 16 December 2023
884:from the original on 22 November 2023
799:from the original on 25 November 2023
632:from the original on 19 December 2023
580:from the original on 19 December 2023
72:
7:
1066:from the original on 4 December 2023
1034:from the original on 9 December 2023
970:from the original on 17 January 2024
910:from the original on 9 December 2023
858:from the original on 4 December 2023
756:from the original on 8 December 2023
693:from the original on 4 December 2023
660:Union Register of medicinal products
512:from the original on 9 December 2023
469:"Summary of Product Characteristics"
324:technology to be approved by the US
151:
999:(Press release). 15 December 2023.
795:(Press release). 16 November 2023.
425:The therapy has a US list price of
269:
966:(Press release). 16 January 2024.
25:
1519:
1022:Feuerstein A (8 December 2023).
766:
590:
440:The UK price is estimated to be
956:"FDA Roundup: January 16, 2024"
931:"FDA Roundup: January 16, 2024"
506:Electronic Medicines Compendium
437:(QALY) at $ 100,000–$ 150,000.
1:
656:"Casgevy product information"
317:thalassemia in January 2024.
1322:Other hematological agents (
1086:"Value Assessment Framework"
962:Food and Drug Administration
818:Sheridan C (November 2023).
748:Food and Drug Administration
572:Food and Drug Administration
382:Food and Drug Administration
326:Food and Drug Administration
285:, sold under the brand name
1249:(Supplement 1): 4899–4901.
681:Stein R (31 October 2023).
1573:
1473:Lovotibeglogene autotemcel
1175:10.1038/d41586-023-03590-6
918:– via Business Wire.
892:– via Business Wire.
836:10.1038/d41587-023-00016-6
435:quality-adjusted life year
297:and transfusion-dependent
293:used for the treatment of
1256:10.1182/blood-2022-166881
1221:10.1182/blood-2022-162353
995:European Medicines Agency
624:European Medicines Agency
415:European Medicines Agency
1468:Exagamglogene autotemcel
1458:Betibeglogene autotemcel
1148:Wong C (November 2023).
322:CRISPR/Cas9 gene editing
283:Exagamglogene autotemcel
115:Exagamglogene autotemcel
34:Exagamglogene autotemcel
1214:(Supplement 1): 29–31.
409:In December 2023, the
303:Vertex Pharmaceuticals
301:. It was developed by
349:vaso-occlusive crises
824:Nature Biotechnology
628:. 14 December 2023.
508:. 24 November 2023.
1557:Sickle-cell disease
1440:sickle cell disease
1362:+desoxyribonuclease
1166:2023Natur.623..676W
752:. 8 December 2023.
576:. 8 December 2023.
400:Society and culture
307:CRISPR Therapeutics
295:sickle cell disease
187:(Prescription only)
36:
1353:Desoxyribonuclease
662:. 12 February 2024
482:on 8 December 2023
431:willingness to pay
1507:
1506:
1160:(7988): 676–677.
541:. 22 January 2024
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1552:Orphan drugs
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1409:C1-inhibitor
1404:Berotralstat
1358:Fibrinolysin
1348:Chymotrypsin
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405:Legal status
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359:Side effects
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332:Medical uses
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170:Legal status
164:Legal status
96:License data
29:
27:Gene therapy
1429:Lanadelumab
1419:Ecallantide
1133:10 December
1102:10 December
1038:10 December
1007:16 December
666:19 February
636:16 December
390:orphan drug
216:Identifiers
133:Intravenous
82:MedlinePlus
55:Other names
46:Trade names
1536:Categories
1391:angioedema
1389:hereditary
1070:4 December
974:17 January
940:19 January
914:9 December
888:9 December
862:4 December
830:(1): 3–4.
803:8 December
760:8 December
697:4 December
584:8 December
516:9 December
486:9 December
448:References
442:£1 million
394:fast track
251:S53L777GM8
1483:Voxelotor
1478:Mitapivat
1424:Icatibant
1343:Bromelain
1332:Enzymes (
1265:256788715
1230:254352365
852:265350318
566:"Casgevy"
421:Economics
338:indicated
126:Routes of
74:Monograph
68:Drugs.com
1526:Medicine
1184:37974039
1127:Archived
1096:Archived
1064:Archived
1032:Archived
1001:Archived
968:Archived
908:Archived
882:Archived
856:Archived
844:37989785
797:Archived
754:Archived
691:Archived
630:Archived
578:Archived
539:DailyMed
510:Archived
222:DrugBank
140:ATC code
110:DailyMed
1378:Trypsin
1162:Bibcode
545:3 March
380:The US
368:History
328:(FDA).
289:, is a
287:Casgevy
231:DB15572
156:)
150: (
148:B06AX05
112::
88:a624018
50:Casgevy
18:Exa-cel
1512:Portal
1492:Others
1263:
1228:
1182:
1154:Nature
850:
842:
793:(MHRA)
433:for 1
271:D12749
198:℞-only
196:
183:
108:
1499:Hemin
1449:B06AX
1395:B06AC
1334:B06AA
1261:S2CID
1243:Blood
1226:S2CID
1208:Blood
997:(EMA)
964:(FDA)
960:U.S.
848:S2CID
750:(FDA)
746:U.S.
626:(EMA)
574:(FDA)
570:U.S.
480:(PDF)
1443:and
1180:PMID
1135:2023
1104:2023
1072:2023
1040:2023
1028:STAT
1009:2023
976:2024
942:2024
916:2023
890:2023
864:2023
840:PMID
805:2023
762:2023
699:2023
668:2024
638:2023
586:2023
547:2024
518:2023
488:2023
305:and
262:KEGG
242:UNII
64:AHFS
1324:B06
1251:doi
1247:140
1216:doi
1212:140
1170:doi
1158:623
935:FDA
832:doi
687:NPR
185:POM
153:WHO
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192:US
179:UK
105:US
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