New hemophilia treatment decision in January with Emicizumab (ACE910) from Chugai and Roche / Genentech
I’ve been following the prospects for emicizumab since hearing about it from a relative in Japan. So it was exciting to see this week’s announcement by Genentech (subsidiary of Roche) that the FDA has granted Priority Review for emicizumab as prophylaxis with a once-weekly subcutaneous treatment for adults and children with hemophilia A. The FDA is expected to make a decision on approval in the next six months.
Since it’s a different pathway than standard hemophilia treatments, emicizumab seems like a very optimistic development. It operates indirectly on the factor viii deficiency by bringing together factors IXa and X to restore the workings of the coagulation cascade. The very significant benefits to this treatment are that it lasts a week and is delivered under the skin.
The reason the FDA granted an expedited approval was due to favorable results from two studies of emicizuma called HAVEN 1 (studying adolescents and adults) and HAVEN 2 (studying children under age 12). The HAVEN 2 study shows that of the 19 children receiving emicizumab there was only 1 treated bleed.
Currently it’s for those with inhibitors only, so we’ll be keeping a close eye on its potential for treating the larger Hemophilia A population.
