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Haemophilia treatment with new delivery method approved
The US FDA has authorised the first treatment for haemophilia A or B that is administered via a pre-filled pen or syringe.
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haemophilia B
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Pfizer receives EU haemophilia B gene therapy approval
The single-dose gene therapy was approved for certain patients with haemophilia B in the US and Canada earlier this year, marketed as BEQVEZ.
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Novel haemophilia B gene therapy approved
Pfizer has attained its first approval for a gene therapy from the US Food and Drug Administration (FDA), which is indicated to treat the rare blood disorder haemophilia B.
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The evolution of AAVs in cell and gene therapy
AAV development for cell and gene therapy in 2023 is being impacted by manufacturing and regulation challenges, however advancing technologies offer opportunity, according to leaders in the field.
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EC approves first gene therapy for haemophilia B
The first gene therapy for haemophilia B has been given conditional marketing authorisation in Europe by the European Commission.
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EMA releases human medicines highlights 2022 report
Medicines which represented significant progress in their therapeutic areas have been listed in the European Medicines Agency's Human Medicines Highlights 2022 report.
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CHMP meeting highlights – December 2022
A total of five drugs were recommended for approval at the CHMP’s December meeting, including a gene therapy and two cancer drugs.
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CHMP adopts positive opinion for haemophilia B gene therapy
Gene therapy etranacogene dezaparvovec has been granted a positive opinion by the CHMP as a haemophilia B treatment for adults in Europe.
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FDA approves first gene therapy for Haemophilia B
Hemgenix, the first gene therapy for adults with Haemophilia B has been approved by the US Food and Drug Administration (FDA).
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Summer 2022 haemophilia trial update
Results from several haemophilia trials have been presented at the International Society on Thrombosis and Haemostasis (ISTH) Annual Congress 2022, here EPR summarises some of the key developments.
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Concizumab haemophilia trials paused over safety concerns
The occurrence of non-fatal thrombotic events in Phase III study participants has called into question the safety of concizumab.
