This page shows the latest Beta thalassemia news and features for those working in and with pharma, biotech and healthcare.
That delays also include its application to the US Food and Drug Administration (FDA) for its gene therapy for beta thalassemia LentiGlobin – which has already been approved in Europe with the
The two patients have severe haemoglobinopathies – including transfusion-dependant beta thalassaemia (TDT) and severe sickle cell disease (SCD). ... beta thalassemia,” said Jeffrey Leiden (pictured left), president and chief executive officer of
Meanwhile Bluebird is proposing a similar five-year instalment plan for its 1.57m beta thalassemia therapy Zynteglo.
US biotech pioneering outcomes-based payment in Europe. Bluebird Bio’s gene therapy treatment for beta thalassemia Zynteglo is to be priced at 1.575m ($1.76m) in Europe, where the ... As the therapy is only currently licensed for use in beta
That’s because Bluebird believes it can offer a compelling cost-effectiveness case for its treatment in beta thalassemia and sickle cell cases versus conventional stem cell treatment. ... beta thalassemia patients live in these four count.
Another gene therapy pioneer, with a similar payment model looks likely to beat Novartis to the European market, however - Bluebird Bio and its beta thalassemia treatment.
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Revenue projection in the year 2025 is $1.259bn,” he continued. Ringheim cited Celgene’s beta thalassemia candidate Sotatercept, which is expected to achieve sales of $458m by 2023, as a
LentiGlobin (betibeglogene darolentivec), an Orphan Drug and Breakthrough Therapy from Bluebird Bio, corrects the defect causing beta thalassemia, a blood disorder that causes life-threatening anaemia. ... The initial studies for LentiGlobin in
Bluebird Bio is just weeks away from gaining its first ever approval for its first ever product, Zynteglo, a groundbreaking gene therapy treatment for beta thalassemia. ... Increasingly factored into this equation is competition from companies following
with sickle cell disease or beta thalassemia with FDA Fast Track Designation.
beta thalassemia – to become the first CRISPR drug to start testing in Europe. ... The FDA placed the CTX001 programmed in beta thalassemia and a planned trial in sickle cell disease on a clinical hold shortly afterwards, but relaxed that last October.
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