406:(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.
362:
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
344:
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
361:
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
305:
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
896:
352:
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.
867:"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"
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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.
892:
785:
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418:. 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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631:
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
1107:
1288:
893:"Vertex and CRISPR Therapeutics Announce US FDA Approval of Casgevy (exagamglogene autotemcel) for the Treatment of Sickle Cell Disease"
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385:, and regenerative medicine advanced therapy designations. The FDA granted approval of Casgevy to Vertex Pharmaceuticals.
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775:"MHRA authorises world-first gene therapy that aims to cure sickle-cell disease and transfusion-dependent β-thalassemia"
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1228:"Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia"
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1193:"Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Severe Sickle Cell Disease"
1191:
Frangoul H, Locatelli F, Bhatia M, Mapara MY, Molinari L, Sharma A, et al. (November 2022).
1013:"In historic decision, FDA approves a CRISPR-based medicine for treatment of sickle cell disease"
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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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979:"First gene editing therapy to treat beta thalassemia and severe sickle cell disease"
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672:"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
1417:
1407:
1043:"ICER Publishes Final Evidence Report on Gene Therapies for Sickle Cell Disease"
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340:, and for the treatment of people with transfusion-dependent beta-thalassemia.
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731:"FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease"
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1139:"UK first to approve CRISPR treatment for diseases: what you need to know"
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1108:"Life-changing blood disease treatment approved at £1m cost per patient"
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809:"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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869:(Press release). Vertex Pharmaceuticals. 16 November 2023.
373:(FDA) granted the application for exagamglogene autotemcel
895:(Press release). Vertex Pharmaceuticals. 8 December 2023.
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but for whom a suitable stem cell donor is not available.
524:"Casgevy- exagamglogene autotemcel injection, suspension"
491:"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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1106:Pinkstone J, Searles M (16 November 2023).
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1080:Institute for Clinical and Economic Review
1048:Institute for Clinical and Economic Review
1270:"Exagamglogene Autotemcel (Code C169042)"
1243:
1208:
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464:(MHRA). 15 November 2023. Archived from
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331:hematopoietic stem cell transplantation
325:In the UK, exagamglogene autotemcel is
218:
75:
20:
1118:from the original on 10 December 2023
1087:from the original on 14 November 2023
992:from the original on 16 December 2023
873:from the original on 22 November 2023
788:from the original on 25 November 2023
621:from the original on 19 December 2023
569:from the original on 19 December 2023
61:
7:
1055:from the original on 4 December 2023
1023:from the original on 9 December 2023
959:from the original on 17 January 2024
899:from the original on 9 December 2023
847:from the original on 4 December 2023
745:from the original on 8 December 2023
682:from the original on 4 December 2023
649:Union Register of medicinal products
501:from the original on 9 December 2023
458:"Summary of Product Characteristics"
313:technology to be approved by the US
140:
988:(Press release). 15 December 2023.
784:(Press release). 16 November 2023.
414:The therapy has a US list price of
258:
955:(Press release). 16 January 2024.
14:
1508:
1011:Feuerstein A (8 December 2023).
755:
579:
429:The UK price is estimated to be
945:"FDA Roundup: January 16, 2024"
920:"FDA Roundup: January 16, 2024"
495:Electronic Medicines Compendium
426:(QALY) at $ 100,000–$ 150,000.
1:
645:"Casgevy product information"
306:thalassemia in January 2024.
1311:Other hematological agents (
1075:"Value Assessment Framework"
951:Food and Drug Administration
807:Sheridan C (November 2023).
737:Food and Drug Administration
561:Food and Drug Administration
371:Food and Drug Administration
315:Food and Drug Administration
274:, sold under the brand name
1238:(Supplement 1): 4899–4901.
670:Stein R (31 October 2023).
1562:
1462:Lovotibeglogene autotemcel
1164:10.1038/d41586-023-03590-6
907:– via Business Wire.
881:– via Business Wire.
825:10.1038/d41587-023-00016-6
424:quality-adjusted life year
286:and transfusion-dependent
282:used for the treatment of
1245:10.1182/blood-2022-166881
1210:10.1182/blood-2022-162353
984:European Medicines Agency
613:European Medicines Agency
404:European Medicines Agency
1457:Exagamglogene autotemcel
1447:Betibeglogene autotemcel
1137:Wong C (November 2023).
311:CRISPR/Cas9 gene editing
272:Exagamglogene autotemcel
104:Exagamglogene autotemcel
23:Exagamglogene autotemcel
1203:(Supplement 1): 29–31.
398:In December 2023, the
292:Vertex Pharmaceuticals
290:. It was developed by
338:vaso-occlusive crises
813:Nature Biotechnology
617:. 14 December 2023.
497:. 24 November 2023.
1546:Sickle-cell disease
1429:sickle cell disease
1351:+desoxyribonuclease
1155:2023Natur.623..676W
741:. 8 December 2023.
565:. 8 December 2023.
389:Society and culture
296:CRISPR Therapeutics
284:sickle cell disease
176:(Prescription only)
25:
1342:Desoxyribonuclease
651:. 12 February 2024
471:on 8 December 2023
420:willingness to pay
1496:
1495:
1149:(7988): 676–677.
530:. 22 January 2024
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1398:C1-inhibitor
1393:Berotralstat
1347:Fibrinolysin
1337:Chymotrypsin
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159:Legal status
153:Legal status
85:License data
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16:Gene therapy
1418:Lanadelumab
1408:Ecallantide
1122:10 December
1091:10 December
1027:10 December
996:16 December
655:19 February
625:16 December
379:orphan drug
205:Identifiers
122:Intravenous
71:MedlinePlus
44:Other names
35:Trade names
1525:Categories
1380:angioedema
1378:hereditary
1059:4 December
963:17 January
929:19 January
903:9 December
877:9 December
851:4 December
819:(1): 3–4.
792:8 December
749:8 December
686:4 December
573:8 December
505:9 December
475:9 December
437:References
431:£1 million
383:fast track
240:S53L777GM8
1472:Voxelotor
1467:Mitapivat
1413:Icatibant
1332:Bromelain
1321:Enzymes (
1254:256788715
1219:254352365
841:265350318
555:"Casgevy"
410:Economics
327:indicated
115:Routes of
63:Monograph
57:Drugs.com
1515:Medicine
1173:37974039
1116:Archived
1085:Archived
1053:Archived
1021:Archived
990:Archived
957:Archived
897:Archived
871:Archived
845:Archived
833:37989785
786:Archived
743:Archived
680:Archived
619:Archived
567:Archived
528:DailyMed
499:Archived
211:DrugBank
129:ATC code
99:DailyMed
1367:Trypsin
1151:Bibcode
534:3 March
369:The US
357:History
317:(FDA).
278:, is a
276:Casgevy
220:DB15572
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139: (
137:B06AX05
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77:a624018
39:Casgevy
1501:Portal
1481:Others
1252:
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1171:
1143:Nature
839:
831:
782:(MHRA)
422:for 1
260:D12749
187:℞-only
185:
172:
97:
1488:Hemin
1438:B06AX
1384:B06AC
1323:B06AA
1250:S2CID
1232:Blood
1215:S2CID
1197:Blood
986:(EMA)
953:(FDA)
949:U.S.
837:S2CID
739:(FDA)
735:U.S.
615:(EMA)
563:(FDA)
559:U.S.
469:(PDF)
1432:and
1169:PMID
1124:2023
1093:2023
1061:2023
1029:2023
1017:STAT
998:2023
965:2024
931:2024
905:2023
879:2023
853:2023
829:PMID
794:2023
751:2023
688:2023
657:2024
627:2023
575:2023
536:2024
507:2023
477:2023
294:and
251:KEGG
231:UNII
53:AHFS
1313:B06
1240:doi
1236:140
1205:doi
1201:140
1159:doi
1147:623
924:FDA
821:doi
676:NPR
174:POM
142:WHO
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