Clinical News
TIF Position Statement: Demanding Access To Existing And Upcoming Gene Therapies For β-Thalassaemia
4 settimane ago
TIF Position Statement: Demanding Access To Existing And Upcoming Gene Therapies For β-Thalassaemia
The Thalassaemia International Federation (TIF) is expressing its grave disappointment over the recent announcement of Bluebird Bio regarding the “wind-down” of its operations in Europe and the exclusive disposal of…
Mitapivat Granted Priority Review By The FDA For Pyruvate Kinase (PK) Deficiency
Agosto 19, 2021
Mitapivat Granted Priority Review By The FDA For Pyruvate Kinase (PK) Deficiency
The U.S. Food and Drug Administration (FDA) has accepted Agios’ New Drug Application (NDA) for Mitapivat for the treatment of adults with pyruvate kinase (PK) deficiency. The NDA was granted…
CDC, FDA Authorize Third Dose Of COVID-19 Vaccines For The Immunocompromised In The US
Agosto 17, 2021
CDC, FDA Authorize Third Dose Of COVID-19 Vaccines For The Immunocompromised In The US
The U.S. Food and Drug Administration (FDA) and the U.S. Centers for Disease Control and Prevention (CDC) have both recommended on August 16 that certain immunocompromised people receive a third…
Global Blood Submits US Application For Expanded Use Of SCD Drug, Oxbryta, In Patients Aged 4-11 Years
Luglio 30, 2021
Global Blood Submits US Application For Expanded Use Of SCD Drug, Oxbryta, In Patients Aged 4-11 Years
Children with sickle cell disease (SCD) as young as age 4 could be prescribed Oxbryta (Voxelotor) if the U.S. Food and Drug Administration (FDA) approves a supplemental new drug application (sNDA) submitted by…
Bluebird Bio Resumes Marketing Of Gene Therapy Zynteglo For β-Thalassaemia In Europe
Luglio 12, 2021
Bluebird Bio Resumes Marketing Of Gene Therapy Zynteglo For β-Thalassaemia In Europe
The European Medicines Agency (EMA)’s safety committee (PRAC) has concluded that there is no evidence Zynteglo causes a blood cancer known as acute myeloid leukaemia (AML). Zynteglo, a gene therapy…
Clinical Trials Update: The June 2021 Edition
Luglio 5, 2021
Clinical Trials Update: The June 2021 Edition
TIF provides you with comprehensive and up-to-date information on developing drugs and therapies for thalassaemia and sickle cell disease (SCD) currently in clinical trials. Our most recent June 2021 update…
Agios Submits New Drug Application to FDA for Mitapivat for Treatment of Adults with Pyruvate Kinase Deficiency
Giugno 22, 2021
Agios Submits New Drug Application to FDA for Mitapivat for Treatment of Adults with Pyruvate Kinase Deficiency
Agios Pharmaceuticals, Inc. announced that it has submitted a New Drug Application (NDA) for Mitapivat to the U.S. Food and Drug Administration (FDA) for the treatment of adults with pyruvate…
TIF Is Actively Participating In The 26th EHA Annual Congress
Giugno 15, 2021
TIF Is Actively Participating In The 26th EHA Annual Congress
TIF is excited to be participating in the 26th EHA Annual Congress, held this year virtually on 09 – 17 June 2021. Ms Lily Cannon, TIF Operations Manager, attended a…
Bluebirdbio Announces The Lifting Of FDA Clinical Hold for Sickle Cell Disease And β-Thalassaemia Studies
Giugno 8, 2021
Bluebirdbio Announces The Lifting Of FDA Clinical Hold for Sickle Cell Disease And β-Thalassaemia Studies
bluebird bio, Inc. today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical holds on the Phase 1/2 HGB-206 and Phase 3 HGB-210 studies of LentiGlobin…
US STUDY: COVID-19 Does Not Pose A Threat To Blood And Transfusion Safety
Giugno 3, 2021
US STUDY: COVID-19 Does Not Pose A Threat To Blood And Transfusion Safety
A new reassuring US study, published recently in the journal Transfusion, suggests that the risk of transmission of SARS-CoV2 through blood transfusion is highly unlikely. For the study, the investigators…