Beta-thalassemia
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Sign up freeTop drugs studied for this disease
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EMA approval · 2023-12-15First gene editing therapy to treat beta thalassemia and severe sickle cell disease
EMA recommended approval of Casgevy, the first gene-editing therapy using CRISPR/Cas9 for transfusion-dependent beta-thalassemia and severe …
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CLINICAL_TRIALS trial_result · 2023-12-14Clinical Trial Update: Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia [Phase 2]
Phase 2 trial updates the safety, tolerability, PK/PD, and preliminary efficacy of VIT-2763 in beta-thalassemia with non-transfusion-depende…
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CLINICAL_TRIALS trial_result · 2023-12-01Clinical Trial Update: Thalidomide and Hydroxyurea Combination in β-Thalassemia Patients [Phase 2, Phase 3]
A phase 2/3 clinical trial evaluating a thalidomide and hydroxyurea combination in patients with beta-thalassemia has completed enrollment a…
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FDA approval · 2023-08-28FDA Approved: REBLOZYL — Supplemental Application (Celgene Corporation)
FDA approved a supplemental application for Reblozyl (luspatercept) by Celgene Corporation. The action concerns updating the product's label…
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CLINICAL_TRIALS trial_result · 2023-06-18Clinical Trial Update: A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype [Phase 3]
Phase 3 trial evaluating LentiGlobin BB305 for transfusion-dependent beta-thalassemia with non β0/β0 genotype completed; results posted in 2…
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CLINICAL_TRIALS trial_result · 2023-06-18Clinical Trial Update: Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia. [Phase 2]
A Phase 2 trial assessing the safety and tolerability of sotatercept (ACE-011) in adults with beta-thalassemia was terminated; results were …
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EMA withdrawal · 2022-11-30EMA Withdrawn: Zynteglo (autologous CD34+ cell enriched population that contains haematopoietic stem cells transduced with lentiviral vector encoding the βA-T87Q-globin gene) — bluebird bio (Netherlands) B.V.
EMA withdrawn Zynteglo, a gene therapy for beta-thalassemia previously granted conditional approval.
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FDA approval · 2022-09-23FDA Approved: DEFERASIROX — Original Application (Teva Pharmaceuticals, Inc.)
FDA approved Teva's Deferasirox as an original generic application, enabling broader access to this iron chelation therapy for patients with…
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CLINICAL_TRIALS trial_result · 2022-09-21Clinical Trial Update: Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl Hydrazone [Phase 2]
Phase 2 clinical trial update on chelation therapy using Pyridoxal Isonicotinoyl Hydrazone for iron overload in anemia due to beta-thalassem…
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EMA approval · 2022-07-14EMA Authorised: Deferiprone Lipomed (Deferiprone) — Lipomed GmbH
EMA authorised Deferiprone Lipomed for iron overload and beta-thalassemia with Lipomed GmbH as marketing authorisation holder.
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CLINICAL_TRIALS trial_result · 2022-06-30Clinical Trial Update: A Study of IMR-687 in Subjects With Beta Thalassemia [Phase 2]
Phase 2 study of IMR-687 in Beta-thalassemia was terminated; results have been posted in 2022.
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CLINICAL_TRIALS trial_result · 2021-11-24Clinical Trial Update: Clinical Experience of Thalidomide in Thalassemic Patients [Phase 2, Phase 3]
Clinical trial update reporting experience with thalidomide in thalassemia patients (Phase 2 and Phase 3; completed).
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CLINICAL_TRIALS trial_result · 2021-11-18Clinical Trial Update: A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia [Phase 2]
Phase 2 trial assessing efficacy and safety of luspatercept in adults with non transfusion dependent beta-thalassemia has completed with res…
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CLINICAL_TRIALS trial_result · 2021-11-12Clinical Trial Update: Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP) [Phase 2]
Phase 2 clinical trial update investigating sirolimus for beta-thalassemia in transfusion-dependent patients.
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EMA safety_alert · 2021-07-23CHMP endorses review finding no link between viral vector in Zynteglo and blood cancer
The CHMP endorses a review finding that there is no link between the viral vector used in Zynteglo and the development of blood cancer.
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CLINICAL_TRIALS trial_result · 2021-07-16Clinical Trial Update: Safety Study for Beta Thalassemia Subjects on PTG-300 [Phase 2]
Phase 2 safety study for PTG-300 in beta-thalassemia subjects completed.
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CLINICAL_TRIALS trial_result · 2021-07-16Clinical Trial Update: Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia [Phase 2]
Phase 2 trial update for PTG-300 in beta-thalassemia subjects with chronic anemia (both transfusion-dependent and non-transfusion-dependent)…
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EMA safety_alert · 2021-07-09EMA finds no evidence linking viral vector in Zynteglo to blood cancer
The EMA reports no evidence that the viral vector used in Zynteglo is linked to blood cancer, though continued monitoring is advised.
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CLINICAL_TRIALS trial_result · 2021-06-07Clinical Trial Update: Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment [Phase 2]
Phase 2 trial update on cardiac T2* in beta-thalassemia patients treated with deferasirox; results posted after completion.
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FDA approval · 2021-04-30FDA Approved: FERRIPROX — Supplemental Application (Chiesi USA, Inc.)
FDA approved a supplemental application for Ferriprox (deferiprone) submitted by Chiesi USA, Inc. This action approves changes to the produc…
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FDA label_change · 2020-07-23FDA Approved: JADENU — Supplemental Application (Novartis Pharmaceuticals Corporation)
FDA approves a supplemental application for JADENU (deferasirox) from Novartis, updating the product labeling.
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CLINICAL_TRIALS trial_result · 2020-05-01Clinical Trial Update: Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO [NA]
Clinical trial update for allogeneic stem cell transplantation in severe beta-thalassemia variants; the study is terminated with results pos…
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FDA approval · 2020-04-03FDA Approved: REBLOZYL — Original Application (Celgene Corporation)
FDA approved Reblozyl (luspatercept) in the original application for adults with transfusion-dependent anemia due to myelodysplastic syndrom…
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CLINICAL_TRIALS trial_result · 2020-03-10Clinical Trial Update: A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease [Phase 1, Phase 2]
A completed early-phase study evaluating the safety and efficacy of LentiGlobin BB305 in transfusion-dependent beta-thalassemia and sickle c…
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FDA approval · 2020-02-20FDA Approved: FERRIPROX — Supplemental Application (Chiesi USA, Inc.)
FDA approved a supplemental application for Ferriprox (deferiprone) submitted by Chiesi USA, Inc.