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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 A
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Haemophilia drug could reduce treated bleeding episodes
Once-weekly or once-monthly dosing of Mim8 provides optionality and flexibility for people living with haemophilia A with or without inhibitors, stated Novo Nordisk’s EVP for Development.
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UK clinical trial regulation may support haemophilia treatment access
The new Notification scheme by the MHRA helps to reduce the time it takes for the lowest-risk clinical trials to commence, while maintaining patient safety.
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CHMP meeting highlights: April 2024
In its April meeting, the EMA’s human medicines committee recommended eight new medicines, including treatments for cancer and autoimmune diseases.
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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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First gene therapy approved for Haemophilia A
The first approved gene therapy for haemophilia A reduced the mean annualised bleeding rate from 5.4 bleeds to 2.6 bleeds per year in a clinical study.
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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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Potential blockbuster drugs to watch in 2023
Lecanemab for Alzheimer's and the first potential gene therapy primed for US launch for haemophilia A are two potential blockbuster Drugs to Watch™, says a 2023 report.
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Europe approves first gene therapy for treatment of haemophilia A
European approval of BioMarin’s ROCTAVIAN (valoctocogene roxaparvovec) one-time gene therapy represents a breakthrough in the treatment of adults with severe haemophilia A.
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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.
![Roche logo sign lit up [Credit: testing/Shutterstock.com].](https://www.europeanpharmaceuticalreview.com/wp-content/uploads/Roche-3-300x278.jpg)
![Roche logo sign lit up [Credit: testing/Shutterstock.com].](https://www.europeanpharmaceuticalreview.com/wp-content/uploads/Roche-3-e1652444508201.jpg)
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Roche announces plans to improve diagnosis and treatments in developing countries
Roche shares initiative commitments that will extend prophylactic treatment access to patients with haemophilia, as well as investments to provide testing capabilities for HIV and Tuberculosis in developing countries.
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Efanesoctocog alfa prevents bleeding episodes in haemophilia A patients
Efanesoctocog alfa significantly and meaningfully reduces annualised bleeding rates in haemophilia A patients, according to new Phase III data.
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Gene therapy sustains bleeding control in haemophilia A trial
Giroctocogene fitelparvovec had a mean annualised bleeding rate (ABR) of just 1.4 percent over two years, with no bleeding events in the first year post infusion.
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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.
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Almost 400 cell and gene therapies in US pipeline, finds report
New research has found that there are 362 cell and gene therapies in clinical pipelines in the US, an increase from 2018.
