A novel haemophilia treatment has passed its first regulatory stage in the UK.
Marstacimab is the first treatment of its kind, and works by targeting the protein TFPI which normally reduces blood clotting ability. It gained European Union approval in November.
Marstacimab, marketed as Hympavzi, has now been approved by the Medicines and Healthcare products Regulatory Agency (MHRA), making it legal to use in the UK. It is currently being evaluated by the National Institute for Health and Care Excellence (NICE), which will determine whether it should be funded for use in the NHS.
The treatment is approved for patients aged 12 or over who must weigh at least 35kg. It can be given as a sub-dermal injection weekly, using a pen, allowing patients to self-administer.
The MHRA said its decision was based upon a year-long study of the treatment involving 116 patients. This suggested it is as effective as routine prophylaxis and that it reduced the bleeding rate.
Julian Beach, MHRA interim director for healthcare quality and access, said: “Keeping patients safe and enabling their access to high quality, safe and effective medical products are key priorities for us.
“This new type of treatment demonstrates our commitment to enabling access to safe, innovative and effective medicines. We’re assured that the appropriate regulatory standards for the approval of this medicine have been met. As with all products, we will keep its safety under close review.”
Source: MHRA
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