Amazing....
$448,000 annual price would actually reduce per-patient costs.
Prophylactic treatment with emicizumab (Hemlibra) could soon replace the current standard treatment for hemophilia A patients without factor VIII inhibitors, and at a largely reduced treatment schedule, results of the manufacturer-sponsored HAVEN 3 and HAVEN 4 trials suggested.
The trials showed that emicizumab prevented or significantly reduced bleeds in these patients. Compared with no prophylaxis, emicizumab every week or 2 weeks yielded 96% and 97% reductions in bleeds requiring treatment (P<0.0001 for both), reported Michael Callaghan, MD, of Children's Hospital of Michigan in Detroit, and colleagues.
In addition, bleeds were reduced by 68% among patients who previously received standard of care factor VIII prophylaxis (P<0.0001).
"In the long run, as long as there aren't any unexpected safety events ... I think this will be the standard of care," Callaghan told MedPage Today.
Results of both trials were presented at the World Federation of Hemophilia's World Congress in Glasgow.
During the study period, 55.6% of patients on the once-weekly emicizumab schedule (95% CI 38.1-72.1) and 60% of those on the every 2-week schedule (95% CI 42.1-76.1) experienced no bleeds. None of the patients randomized to no prophylaxis were free from bleeds.
Callaghan said he did not see many hurdles in terms of emicizumab becoming the preferred first-line therapy for hemophilia A. While the price of the drug is high, annual cost-effectiveness estimates place it lower than current factor VIII prophylaxis.