FDA expands Roche hemophilia med approval

Oct. 7, 2018

Roche announced that the U.S. Food and Drug Administration has expanded the use its hemophilia drug Hemlibra to include almost all patients with hemophilia A. The drug has been approved to treat adults, children and newborns with the genetic disorder.

Hemlibra can now be used for patients who have not become resistant to standard treatment. It is the only medicine that can be self-administered subcutaneously once weekly, every two weeks or every four weeks for haemophilia A with and without factor VIII inhibitors. It is also the first medicine to significantly reduce treated bleeds compared to prior factor VIII prophylaxis based on an intra-patient comparison.

Hemlibra won initial U.S. approval last year only for hemophilia A patients with factor VIII resistance, or inhibitors. That accounted for only about 5 percent to 7 percent of the estimated 16,000 U.S. hemophilia A patients.

Read the full Roche release