About 50,000 boys and men in the US and Europe are living with haemophilia A, a congenital disease that primarily affects boys and men. The treatment for patients with the severe form of haemophilia A is factor VIII replacement therapy. About 30 percent of the patients treated with factor VIII develop inhibitory antibodies, making the treatment ineffective. This complication can often be managed with regimens to induce immune tolerance, involving frequent injections of higher doses of factor VIII. The treatment is costly and may last for one or two years. The antibodies remain in about 30 percent of these “inhibitor patients”, leaving them unable to prevent bleeding. Inhibitor patients are being treated with agents with only a short duration of effect, at a cost of as much as SEK 3-8 million per patient a year, to stop acute bleeds.
A new and effective drug for this patient group will soon be launched in the US. One-year treatment costs are believed to be almost SEK 4 million, and analysts estimate that the preparation can reach annual sales of more than SEK 17 billion. However, this drug is associated with the risk of severe side effects, and treatment is lifelong. There are still, therefore, major unmet needs for safer, more cost-effective therapies.