NICE rejection for CSL Behring gene therapy
Therapy is a treatment option for adults with severe and moderately severe haemophilia B
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Tag: haemophilia B
UniQure’s gene therapy AMT-061 looking promising mid trial
UniQure’s experimental gene therapy has shown sustained Factor IX activity in a Phase II study assessing its potential to treat haemophilia B.
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Pfizer kicks off PhIII programme for haemophilia B gene therapy
Pfizer has kicked off a late-stage trial assessing efficacy and safety of the investigational gene therapy fidanacogene elaparvovec for the treatment of haemophilia B.
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Novo’s Refixia beats rival in head to head trial
Novo Nordisk’s Refixia has come out on top in a head to head study assessing its effect in patients with the bleeding disorder haemophilia B against Swedish Orphan Biovitrum’s recombinant factor IX-Fc fusion protein (rFIXFc).
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EU nod for Novo’s long-acting haemophilia drug
Novo Nordisk says it expects to launch its long-acting haemophilia B drug Refixia in the European Union in the fourth quarter of this year after receiving a green light from regulators.
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UniQure’s haemophilia B therapy picks up PRIME status
uniQure’s investigational gene therapy for patients with the blood clotting disorder haemophilia B has been awarded PRIME status by European regulators, potentially placing the treatment on a faster path to approval.
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Six new medicines backed for EU approval
Six new medicines, including three orphan drugs for rare diseases, have been backed for European Union approval by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP).
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Breakthrough status for Pfizer/Spark haemophilia gene therapy
Pfizer and Spark Therapeutics say their investigational haemophilia B drug SPK-9001 has been awarded breakthrough therapy status by the US Food and Drug Administration.
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