The European Medicines Agency Review of Luspatercept for the Treatment of Adult Patients With Transfusion-dependent Anemia Caused by Low-risk Myelodysplastic Syndromes With Ring Sideroblasts or Beta-thalassemia.

Autor: Delgado J; Oncology and Haematology Office, European Medicines Agency, Amsterdam, The Netherlands.; Department of Haematology, Hospital Clinic, Barcelona, Spain., Voltz C; Oncology and Haematology Office, European Medicines Agency, Amsterdam, The Netherlands., Stain M; Bundesamt fur Sicherheit im Gesundheitswesen, Vienna, Austria.; Committe for Medicinal Products for Human Use, European Medicines Agency, Amsterdam, The Netherlands., Balkowiec-Iskra E; Committe for Medicinal Products for Human Use, European Medicines Agency, Amsterdam, The Netherlands.; Urząd Rejestracji Produktów Leczniczych, Wyrobow Medycznych i Produktów Biobójczych, Warsaw, Poland.; Department of Experimental and Clinical Pharmacology, Medical University of Warsaw, Poland., Mueller B; Bundesamt fur Sicherheit im Gesundheitswesen, Vienna, Austria., Wernsperger J; Bundesamt fur Sicherheit im Gesundheitswesen, Vienna, Austria., Malinowska I; Urząd Rejestracji Produktów Leczniczych, Wyrobow Medycznych i Produktów Biobójczych, Warsaw, Poland.; Department of Paediatrics, Haematology and Oncology, Medical University of Warsaw, Warsaw, Poland., Gisselbrecht C; Department of Haematology, Hopital Saint Louis, Paris, France., Enzmann H; Committe for Medicinal Products for Human Use, European Medicines Agency, Amsterdam, The Netherlands.; Bundesinstitut für Arzneimittel und Medizinprodukte, Bonn, Germany., Pignatti F; Oncology and Haematology Office, European Medicines Agency, Amsterdam, The Netherlands.
Jazyk: angličtina
Zdroj: HemaSphere [Hemasphere] 2021 Jul 19; Vol. 5 (8), pp. e616. Date of Electronic Publication: 2021 Jul 19 (Print Publication: 2021).
DOI: 10.1097/HS9.0000000000000616
Abstrakt: Luspatercept is a recombinant fusion protein that selectively binds to ligands belonging to the transforming growth factor-beta superfamily, resulting in erythroid maturation and differentiation. On June 25, 2020, a marketing authorization valid through the European Union (EU) was issued for luspatercept for the treatment of adult patients with transfusion-dependent anemia caused by very low-, low-, and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, or those with transfusion-dependent beta thalassemia (BT). Luspatercept was evaluated in 2 separate phase 3, double-blind, placebo-controlled multicentre trials. The primary endpoints of these trials were the percentage of patients achieving transfusion independence over ≥8 weeks or longer for patients with MDS, and the percentage of patients achieving a ≥33% reduction in transfusion burden from baseline to week 13-24 for patients with BT. In the MDS trial, the percentage of responders was 37.91% versus 13.16%, P < 0.0001, for patients receiving luspatercept versus placebo, respectively. In the BT trial, the percentage of responders was 21.4% versus 4.5% ( P < 0.0001) for luspatercept versus placebo, respectively. Treatment with luspatercept led to similar incidences of adverse events (AEs), but higher incidences of grade ≥3 AEs and serious AEs compared to placebo. The most frequently reported treatment-emergent AEs (≥15%) in the pooled luspatercept group were headache; back pain, bone pain, and arthralgia; diarrhea; fatigue; pyrexia; and cough. The aim of this article is to summarize the scientific review of the application, which led to the regulatory approval in the EU.
(Copyright © 2021 the Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the European Hematology Association.)
Databáze: MEDLINE
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