A new experimental haemophilia treatment from Pfizer has showed promise in reducing patients’ bleeding rates, the company said this week, with a late stage trial showing superiority to the current standard of care treatment.
The therapy, marstacimab, met its main trial goal, demonstrating superiority to factor replacement therapies, with a 92% reduction in bleeding in patients with severe haemophilia A and moderately severe to severe haemophilia B.
Haemophilia hampers the body’s ability to make blood-clotting proteins, leading to prolonged bleeding after injuries or surgery, and primarily affects males, reports Reuters.
In the US, it is estimated there are 12 cases per 100 000 males for haemophilia A and 3.7 cases per 100 000 males for haemophilia B.
The current standard of care requires regular infusion of the missing proteins.
Pfizer’s once weekly marstacimab is an anti-tissue factor pathway inhibitor that could help initiate blood clotting.
Australian drugmaker CSL Ltd’s gene therapy Hemgenix offers a long-term solution for haemophilia B patients, but is among the world’s most expensive treatments.
Pfizer is developing at least two other therapies for haemophilia: in December, the company met its main goal in a late-stage study for a haemophilia B gene therapy.
The current trial of marstacimab only tested patients who did not have any antibodies that stop the production of the blood-clotting proteins, called factor VIII or factor IX.
Pfizer is also testing marstacimab in patients with those inhibitors, with data expected in late 2024.
Reuters article – Pfizer’s hemophilia therapy reduces bleeding in late-stage study (Open access)
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