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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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.
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.
The first gene therapy for haemophilia B has been given conditional marketing authorisation in Europe by the European Commission.
Medicines which represented significant progress in their therapeutic areas have been listed in the European Medicines Agency's Human Medicines Highlights 2022 report.
A total of five drugs were recommended for approval at the CHMP’s December meeting, including a gene therapy and two cancer drugs.
Gene therapy etranacogene dezaparvovec has been granted a positive opinion by the CHMP as a haemophilia B treatment for adults in Europe.
Hemgenix, the first gene therapy for adults with Haemophilia B has been approved by the US Food and Drug Administration (FDA).
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.
The occurrence of non-fatal thrombotic events in Phase III study participants has called into question the safety of concizumab.