Clinical News

Oxbryta Gets Marketing Authorisation In The UK For Patients With Sickle Cell Disease
Clinical News

Oxbryta Gets Marketing Authorisation In The UK For Patients With Sickle Cell Disease

Voxelotor, an oral treatment taken once daily, is the first medicine authorized in Great Britain that directly inhibits sickle haemoglobin (HbS) polymerization, the molecular basis of sickling and destruction of…
SCIENTIFIC NEWS | Human Trials Of Universal Flu And COVID-19 Vaccines Begin
Clinical News

SCIENTIFIC NEWS | Human Trials Of Universal Flu And COVID-19 Vaccines Begin

  The National Institutes of Health’s (NIH) work on a universal flu vaccine is already well advanced, with human trials beginning now. The new formulation, called BPL-1357, contains a variety…
Mitapivat Receives FDA Approval For Haemolytic Anaemia From Pyruvate Kinase Deficiency
Clinical News

Mitapivat Receives FDA Approval For Haemolytic Anaemia From Pyruvate Kinase Deficiency

The US Food and Drug Administration (FDA) has announced the approval of Mitapivat (PYRUKYND®) tablets as treatment for haemolytic anemia in adults with pyruvate kinase (PK) deficiency. The oral PK…
What’s New In Clinical Trials For Thalassaemia/Sickle Cell Disease Drugs And Therapies?
Clinical News

What’s New In Clinical Trials For Thalassaemia/Sickle Cell Disease Drugs And Therapies?

Many pharmaceutical companies are making remarkable progress in their research into candidate treatments for these diseases and 2022 is expected to be yet another eventful year for haemoglobin disorders! Check…
FDA Grants Priority Review to Luspatercept-aamt for Anemia in Adults With NTD β-Thalassaemia
Clinical News

FDA Grants Priority Review to Luspatercept-aamt for Anemia in Adults With NTD β-Thalassaemia

The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2022. In addition, the European Medicines Agency (EMA) has validated the Type II variation…
TIF Releases New Scientific Bulletin To Highlight Research On Haemoglobin Disorders
Clinical News

TIF Releases New Scientific Bulletin To Highlight Research On Haemoglobin Disorders

A key objective of the Thalassaemia International Federation is to inform and update regularly the global haemoglobinopathies family, both patients and physicians, on the progress and new developments that arise…
UK & Wales: NICE Recommends First Treatment In Two Decades For Sickle Cell Disease
Clinical News

UK & Wales: NICE Recommends First Treatment In Two Decades For Sickle Cell Disease

For the first time in 20 years, a new therapy for sickle cell disease is to be made available on the UK National Health Service (NHS). Crizanlizumab (Adakveo) by Novartis…
Mitapivat Granted Priority Review By The FDA For Pyruvate Kinase (PK) Deficiency
Clinical News

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…
Global Blood Submits US Application For Expanded Use Of SCD Drug, Oxbryta, In Patients Aged 4-11 Years
Clinical News

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…
Clinical Trials Update: The June 2021 Edition
Clinical News

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…
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