Nova’s Xromi approved in UK and EU for vaso-occlusive complications of SCD
The inherited disorder is caused by genetic mutations that affects haemoglobin production
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Tag: sickle cell disease
NHS to test new blood group genotyping test on inherited blood disorder patients
Patients living with sickle cell disease and thalassaemia will be eligible for the test
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EU regulator recommends revoking approval for Novartis’ sickle cell disease drug
The EMA’s human medicines committee concluded that the benefits of Adakveo did not outweigh its risks
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bluebird bio’s sickle cell gene therapy granted MHRA ‘innovation passport’
Innovation passport designation scheme aims to reduce the time to market for promising treatments
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EMA begins review of GBT’s sickle cell treatment Oxbryta
GBT is aiming for the approval of Oxbryta to treat haemolytic anaemia in patients with sickle cell disease
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Vertex, CRISPR Therapeutics’ sickle cell gene therapy wins PRIME designation
European Medicines Agency grants designation based on phase I/II data
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Novartis’ crizanlizumab granted FDA Breakthrough Therapy
US regulators have granted Novartis’ crizanlizumab (SEG101) Breakthrough Therapy designation for the prevention of vaso-occlusive crises in patients with sickle cell disease.
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Novartis crizanlizumab shows promise in sickle cell anaemia
A new analysis has shown that Novartis’ crizanlizumab reduced the number of patients with sickle cell disease experiencing a pain crisis.
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Roche signs inclacumab deal with GBT
Roche has sold off rights to inclacumab to Global Blood Therapeutics, which intends to develop the treatment for vaso-occlusive crises (VOC) in patients with sickle cell disease (SCD).
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Novo buys into EpiDestiny’s experimental sickle cell therapy
Danish drugmaker Novo Nordisk has bought a global licence for EpiDestiny’s sickle cell disease (SCD) programme, EPI01.
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Experimental sickle cell disease drug gets PRIME status
Global Blood Therapeutics’ GBT440 has become the first potential treatment for sickle cell disease to be accepted onto the European Medicines Agency’s regulatory support programme PRIME.
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