Clinical News
-
News
CLINICAL NEWS | PYRUKYND® Receives Positive CHMP Opinion For The Treatment of PKD in Adults
PYRUKYND® is a first-in-class, oral PK activator that was recently approved by the U.S. Food and Drug Administration (FDA). If approved by the European Commission (EC), PYRUKYND® will be the first approved disease-modifying therapy…
Weiterlesen » -
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…
Weiterlesen » -
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…
Weiterlesen » -
News
PYRUVATE KINASE DEFICIENCY | FDA Approves First Drug For The Rare Inherited Anemia
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…
Weiterlesen » -
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…
Weiterlesen » -
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…
Weiterlesen » -
News
MITAPIVAT | FDA Grants Priority Review To New Drug For Pyruvate Kinase Deficiency
The NDA was granted a Priority Review designation and has been given a Prescription Drug User Fee Act (PDUFA) action date of February 17, 2022, accelerating the review time from…
Weiterlesen » -
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…
Weiterlesen » -
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…
Weiterlesen » -
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…
Weiterlesen »