Clinical News

Mitapivat Receives FDA Approval For Haemolytic Anaemia From Pyruvate Kinase Deficiency

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?

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…
SCD Drug Oxbryta Receives CHMP Positive Opinion For Approval In The EU

SCD Drug Oxbryta Receives CHMP Positive Opinion For Approval In The EU

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization for Oxbryta® (voxelotor) tablets for the treatment of hemolytic…
FDA Grants Priority Review to Luspatercept-aamt for Anemia in Adults With NTD β-Thalassaemia

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…
La Repubblica: Gene Editing Cured Two Siblings With Thalassaemia In Italy

La Repubblica: Gene Editing Cured Two Siblings With Thalassaemia In Italy

Cured of thalassemia, thanks to gene editing. According to a recent news report published in the Italian newspaper La Repubblica, Erika and Emanuele Guarini, two siblings from Pistoia, Tuscany, aged…
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…
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