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Table 2 Summary and comparison of novel therapies discussed, outlining mechanism of action, route and frequency of administration, advantages, limitations, and the current licensing status

From: Advances in the management of haemophilia: emerging treatments and their mechanisms

Therapy Mechanism of action Route of administration Frequency of administration Advantages Limitations Licensing status
Emicizumab Bispecific antibody mimicking co-factor function of FVIII Subcutaneous Once a week,
Twice a week, or
Monthly
Can be used in patients with FVIII-inhibitors
No need for peripheral venous access
Reduced frequency of administration
Reduced cost of treatment
No need for routine laboratory monitoring, practical to use
Good safety profile
Interferes with the assays used in laboratory monitoring
Insufficient to treat large bleeds on its own, additional haemostatic measures required
Licensed for use in HA patients with and without inhibitors
Fitusiran GalNAc-siRNA conjugate Subcutaneous Once monthly Application in both HA and HB patients with and without inhibitors
Reduced frequency of administration
No need for peripheral venous access
Good safety profile
More research required for dose selection and management of breakthrough bleeds
Lack of paediatric trial data
In phase III of development
Concizumab Anti-TFPI monoclonal antibody Subcutaneous Once daily Application in both HA and HB patients with and without inhibitors
No need for peripheral venous access
Good safety profile
Improvement in patient QoL
Daily administration
Further research required into therapeutic monitoring
Further research required into implications for surgery
In phase III of development
Gene therapy Transduction of a gene coding for deficient factor into patient hepatocytes Intravenous Single dose Application in both HA and HB patients
Reduced frequency of administration
Improvement in patient QoL
Potential role for immune tolerance induction
Lack of long-term follow up
Further investigations required into a wider range of patient demographics
Potential for degradation by anti-capsid antibodies
Diminishing efficacy over time
Undergoing clinical trials
  1. FVIII factor VIII; HA haemophilia A; GalNAc-siRNA N-acetylgalactosamine-small interfering RNA; HB haemophilia B; TFPI tissue factor pathway inhibitor; QoL quality of life