Clinical News

TIF Position Statement: Demanding Access To Existing And Upcoming Gene Therapies For β-Thalassaemia

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

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

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

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

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

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

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

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

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

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…
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