Clinical News

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…
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…
Bluebirdbio Announces The Lifting Of FDA Clinical Hold for Sickle Cell Disease And β-Thalassaemia Studies
Clinical News

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…
The Lancet: Russian Coronavirus Sputnik V Vaccine Achieves Over 90% Efficacy
Clinical News

The Lancet: Russian Coronavirus Sputnik V Vaccine Achieves Over 90% Efficacy

Russia’s Sputnik V vaccine was 91.6% effective in preventing people from developing Covid-19, according to peer-reviewed results from its late-stage clinical trial published yesterday in The Lancet international medical journal.…
Clinical Trial Cleared for GPH101: The First Potentially Curative Sickle Cell Disease Therapy
Clinical News

Clinical Trial Cleared for GPH101: The First Potentially Curative Sickle Cell Disease Therapy

The U.S. Food & Drug Administration (FDA) has cleared an investigational new drug (IND) application for the experimental gene editing therapy GPH101 by Graphite Bio to initiate a Phase 1/2…
What’s New In Clinical Trials for Thalassaemia and Sickle Cell Disease?
Clinical News

What’s New In Clinical Trials for Thalassaemia and Sickle Cell Disease?

There is a lot of hustle and bustle in the scientific fields of thalassemia and sickle cell disease during the past few months, with many companies making remarkable progress in…
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