Note: This story was updated Feb. 24, 2023, to correct the secondary headline since there are several other recombinant factor VIII therapies available. Altuviiio is a first-in-class therapy, but not the first. Efanesoctocog alfa, Sobi and Sanofi’s first-in-class, long-lasting recombinant or man-made factor…
News
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…
Hemophilia A patients at a single center in France reported high rates of satisfaction with Hemlibra (emicizumab) after a year of real-world use, a study has found. Patients reported improvements in health and life quality, as well as reductions in pain and bleeding with the therapy, but problems…
Teens and young adults with moderate and severe hemophilia A on continuous preventive treatment show levels of physical activity similar to young people without the disease, but less than half of teenagers met the weekly physical activity levels recommended by the World Health organization (WHO), a study reports. “We…
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…
Pain, depression, and anxiety medications are used more frequently by hemophilia patients than by the general population, according to an 11-year study across Nordic countries. However, the use of these medications in people with hemophilia was not found to be associated with their use of factor replacement therapies.
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…
High levels of antibodies against clotting factor VIII (FVIII) were associated with a slower response to immunosuppressive treatments in a small group of people with acquired hemophilia A in new study in China. Of 34 patients, 29 achieved a complete response to their first-line immunosuppressive therapy. Still, a lower…
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…
An investigational factor VIIa (FVIIa) therapy called GEN-0828 may be able to minimize blood loss and accelerate blood clotting at lower doses than NovoSeven, an approved medication used to control bleeding in hemophilia patients, according to a study in a mouse model of hemophilia B. Still, both treatments…
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