EU Approves Hemlibra for Moderate Hemophilia A
The decision follows last year's recommendation by CHMP
The European Commission has approved Hemlibra (emicizumab) as a routine preventive treatment for people with moderate hemophilia A without inhibitors.
“We welcome the European Commission’s decision to approve Hemlibra also for people with moderate hemophilia A in the EU,” said Levi Garraway, MD, PhD, Roche’s chief medical officer and head of global product development, in a press release. “As its benefit expands to broader populations, we remain committed to determining how Hemlibra might help even more people with hemophilia A to live a bleed-free life.”
The approval follows last year’s recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP).
In hemophilia A, a protein essential for blood clotting called factor VIII (FVIII) is missing or defective. A lack of FVIII can lead to prolonged and excessive bleeding episodes, particularly in the joints and muscles, causing pain, joint damage, reduced mobility, and a diminished quality of life.
Approved in more than 110 countries, Hemlibra mimics the action of FVIII and prevents or reduces the frequency of bleeding episodes in people with or without inhibitors, which are neutralizing antibodies generated against FVIII that can reduce the effectiveness of treatment.
The therapy had been only approved in the EU for patients with severe disease — those having less than 1% of residual FVIII activity. People with moderate hemophilia retain between 1-5% FVIII activity, but about 85% of them still have recurrent bleeds and one-third have long-term joint problems.
The approval of Hemlibra’s label expansion was supported by data from the Phase 3 HAVEN 6 study (NCT04158648) along with real-world evidence.
Even “moderate disease can produce bleeds that cause irreversible joint damage and impact quality of life,” Garraway said.
The first analysis of the trial was based on data from 72 patients without inhibitors (three women, 69 men), most of whom had moderate hemophilia A (70.8%). Eligible participants reported more than 10 bleeds a year.
All the patients received 3 mg/kg of Hemlibra, given as an under-the-skin injection, once a week for four weeks, followed by either 1.5 mg/kg every week, 3 mg/kg every two weeks, or 6 mg/kg every four weeks. Patients were followed for just over a year (a median of 55.6 weeks).
Data showed 66.7% of Hemlibra-treated patients had no bleeding episodes that required treatment, while 81.9% had no spontaneous bleeds requiring treatment, and 88.9% had no joint bleeds requiring treatment.
No new safety concerns were reported in patients with moderate hemophilia A without inhibitors. Injection site reactions were the most common side effect, reported in 16.7% of participants.