Month: April 2018

April 25, 2018
Emicizumab in patients without inhibitors

The Food and Drug Administration, the US drug regulatory authority, has granted ‘breakthrough therapy designation’ to Roche’s emicizumab (Hemlibra) to support its development as a treatment for patients with haemophilia A who do not have inhibitors. At present, emicizumab is licensed only for patients with inhibitors. A breakthrough therapy receives accelerated review and development through …

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Switch to EHL factors costly in US

An analysis by a US health economics firms has found that switching from conventional to extended half-life clotting factors is associated with a doubling of healthcare costs. Prime Therapeutics manages health plans for US programmes like Medicare and Medicaid ( It analysed the costs associated with the switch for 34 people with haemophilia A and …

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New evidence of the impact of haemophilia on individuals and carers

Two new reports have confirmed how common pain and psychiatric morbidity are among people with haemophilia. B-HERO-S is a US study of 299 adults with haemophilia B and 150 carers which collected data by online survey (Eur J Haematol 2018; Mar 5. doi: 10.1111/ejh.13055). The prevalence of mild, moderate and severe haemophilia was 25%, 60% …

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Factor storage affects patient satisfaction

Clinicians should remind patients that clotting factors don’t generally need to be stored in the fridge because, a study from Italy suggests, this may influence their satisfaction with treatment (Pat Pref Adherence 2018;12:431-41). The survey of 200 people with moderate or severe haemophilia A in seven countries (including the UK) determined how product features affected …

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Novel biotechnology recognised with special EU status

A technology that could offer people with haemophilia B an implant providing long term Factor IX synthesis has been awarded Advanced Therapy Medicinal Product (ATMP) status by the European Medicines Agency’s Committee for Advanced Therapies. ATMPs are medicines for human use that are based on genes, tissues or cells that ‘offer ground breaking new opportunities …

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