Tabelecleucel: Phê duyệt đầu tiên

Springer Science and Business Media LLC - Tập 27 - Trang 425-431 - 2023
Susan J. Keam1
1Springer Nature, Auckland, New Zealand

Tóm tắt

Tabelecleucel (EbvalloTM) là một liệu pháp miễn dịch tế bào T đặc hiệu với virus Epstein-Barr (EBV) đồng loại, nhắm mục tiêu và loại bỏ các tế bào dương tính với EBV (EBV+) theo cách hạn chế bởi kháng nguyên leukocyte ở người (HLA). Tabelecleucel đã được phát triển bởi Atara Biotherapeutics dưới giấy phép từ Trung tâm Ung thư Memorial Sloan-Kettering (MSKCC) để điều trị các rối loạn tăng sinh bạch cầu (LPDs), bao gồm bệnh tăng sinh lympho hậu ghép dương tính với EBV (PTLD) kháng thuốc/ tái phát sau khi điều trị bằng rituximab. Tabelecleucel đã được cấp phép tiếp thị trong 'các trường hợp đặc biệt' vào ngày 16 tháng 12 năm 2022 với tư cách liệu pháp đơn trị cho việc điều trị bệnh nhân người lớn và trẻ em từ 2 tuổi trở lên bị PTLD dương tính với EBV tái phát hoặc kháng trị, những người đã nhận ít nhất một liệu pháp trước đó. Đối với bệnh nhân ghép tạng rắn, liệu pháp trước đó bao gồm hóa trị, trừ khi hóa trị không phù hợp. Bài viết này tóm tắt các mốc quan trọng trong quá trình phát triển tabelecleucel dẫn đến phê duyệt đầu tiên cho PTLD dương tính với EBV.

Từ khóa

#Tabelecleucel #liệu pháp miễn dịch #virus Epstein-Barr #bệnh tăng sinh lympho #phê duyệt thuốc

Tài liệu tham khảo

Dierickx D, Pociupany M, Natkunam Y. Epstein-Barr virus-associated posttransplant lymphoproliferative disorders: new insights in pathogenesis, classification and treatment. Curr Opin Oncol. 2022;34(5):413–21. Ohmoto A, Fuji S. Clinical features and treatment strategies for post-transplant and iatrogenic immunodeficiency-associated lymphoproliferative disorders. Blood Rev. 2021;49: 100807. Lv K, Yin T, Yu M, et al. Treatment advances in EBV related lymphoproliferative diseases. Front Oncol. 2022;12: 838817. Soldan SS, Messick TE, Lieberman PM. Therapeutic approaches to Epstein-Barr virus cancers. Curr Opin Virol. 2022;56: 101260. European Medicines Agency. Tabelecleucel: EPAR—public assessment report. 2022. https://www.ema.europa.eu/. Accessed 22 Jan 2023. Dharnidharka V, Thirumalai D, Jaeger U, et al. Clinical outcomes of solid organ transplant patients with Epstein-Barr virus-driven (EBV+) post-transplant lymphoproliferative disorder (PTLD) who fail rituximab plus chemotherapy: a multinational, retrospective chart review study [abstract no. 2528]. Blood. 2021;138(Suppl 1):2528. Sanz J, Storek J, Socié G, et al. Clinical outcomes of patients with Epstein-Barr virus-driven post-transplant lymphoproliferative disease following hematopoietic stem cell transplantation who fail rituximab: a multinational, retrospective chart review study [abstract no. 1454]. Blood. 2021;138(Suppl 1):1454. European Medicines Agency. Tabelecleucel: EU prescribing information 2022. https://www.ema.europa.eu/. Accessed 23 Jan 2023. Atara Biotherapeutics Inc. Atara Biotherapeutics’ Ebvallo™ (tabelecleucel) receives European Commission Approval as first ever therapy for adults and children with EBV+ PTLD [media release]. 19 Dec 2022. https://investors.atarabio.com/. Atara Biotherapeutics Inc. Atara Biotherapeutics to receive additional near-term milestone payment under updated tabelecleucel (tab-cel®) commercialization agreement with Pierre Fabre [media release]. 28 Sep 2022. https://www.atarabio.com/. Atara Biotherapeutics inc. Atara Biotherapeutics and Pierre Fabre enter strategic collaboration to commercialize tabelecleucel (tab-cel®) [media release]. 4 Nov 2021. https://www.atarabio.com/. Atara Biotherapeutics Inc. Form 10-K. 2022. https://investors.atarabio.com/. Accessed 20 Jan 2023. Atara Biotherapeutics Inc. Atara Biotherapeutics and QIMR Berghofer Medical Research Institute announce exclusive license and research agreements [media release]. 21 Oct 2015. https://www.atarabio.com/. Atara Biotherapeutics Inc. Atara Biotherapeutics announces immunotherapy alliance with Memorial Sloan Kettering Cancer Center [media release]. 23 Sep 2014. https://www.atarabio.com/. Atara Biotherapeutics Inc. Atara Biotherapeutics exercises exclusive license to T-cell technology from Memorial Sloan Kettering Cancer Center [media release]. 15 Jun 2015. https://www.atarabio.com/. Prockop S, Doubrovina E, Suser S, et al. Off-the-shelf EBV-specific T cell immunotherapy for rituximab-refractory EBV-associated lymphoma following transplantation. J Clin Invest. 2020;130(2):733–47. Mahadeo KM, Baiocchi R, Beitinjaneh A, et al. New and updated results from a multicenter, open-label, global phase 3 study of tabelecleucel (tab-cel) for Epstein-Barr virus-positive post-transplant lymphoproliferative disease (EBV+ PTLD) following allogeneic hematopoietic cell (HCT) or solid organ transplant (SOT) after failure of rituximab or rituximab and chemotherapy (ALLELE) [abstract no. 4658]. Blood. 2022;140(Suppl 1):10374–6. Nikiforow S, Baiocchi R, Nasta S, et al. Clinical experience of tabelecleucel in patients with EBV+ primary (PID) or acquired immunodeficiency (AID) associated lymphoproliferative disease [abstract no. 1658]. In: 62nd Annual Meeting and Exposition of the American Society of Hematology. 2020. Prockop SE, Reshef R, Tsai DE, et al. Long-term outcomes of patients with Epstein-Barr virus-driven post-transplant lymphoproliferative disease following solid organ transplant or allogeneic hematopoietic cell transplant treated with tabelecleucel in a multicenter expanded access program study [abstract no. 81]. Biol Blood Marrow Transplant. 2020;26(3 Suppl):S61–2. Prockop S, Gamelin L, Dinavahi R, et al. Overall survival by best overall response with tabelecleucel in patients with Epstein-Barr virus-driven post-transplant lymphoproliferative disease following solid organ or allogeneic hematopoietic cell transplant [abstract no. 887]. Blood. 2021;138(Suppl 1):887. Choquet S, Chaganti S, Uttenthal B, et al. Demographics and treatment outcomes in patients with EBV+ PTLD treated with off-the-shelf EBV-specific CTL under an ongoing expanded access program in Europe: first analyses [abstract no. 7530]. J Clin Oncol. 2022;40(16 Suppl):7530. Jiménez-Kurlander L, Behr G, Bunin N, et al. Updated efficacy and safety of tabelecleucel in patients with Epstein-Barr virus-positive (EBV+) leiomyosarcomas (LMS) [abstract no. 3349]. Blood. 2022;140(Suppl 1):7524–6. Prockop S, Dinavahi R, Navarro W, et al. A multicenter, multicohort, open-label, single-arm per cohort, phase II study to assess the efficacy and safety of tabelecleucel in patients with EBV-associated diseases using an adaptive two-stage study design [abstract no. 2551]. In: 62nd Annual Meeting and Exposition of the American Society of Hematology. 2020.