News

EU approves Hemgenix, one-time gene therapy for hemophilia B

The European Commission has conditionally approved Hemgenix (etranacogene dezaparvovec), a one-time gene therapy to treat adults with hemophilia B. Hemgenix, originally developed by uniQure, is approved for adults with severe and moderately severe hemophilia B without a history of inhibitors. It’s the first gene therapy for…

Certain immune system proteins found to drive FVIII inhibitors

Researchers have identified immune system proteins that might drive the development of inhibitors, or neutralizing antibodies, against factor VIII (FVIII) replacement therapies in people with hemophilia A. These proteins, called complement proteins, appear to work collaboratively with danger signals that alert the body to critical situations in order to…

EU Approves Hemlibra for Moderate Hemophilia A

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…

Efanesoctocog Alfa Reduces Bleeding, Pain in Phase 3 Study

Most people with severe hemophilia A who were given once-weekly treatment with efanesoctocog alfa in the Phase 3 XTEND-1 trial were bleed-free over a year on treatment. The therapy also improved joint and overall physical health and eased pain intensity in the participants compared with previous treatments. Results…