News

For the first time, as part of a U.S. clinical trial, the cell therapy BE-101 has been administered to someone with hemophilia B. Be Biopharma, the company developing BE-101, announced the dosing of the first participant in a first-in-human Phase 1/2 clinical trial called BeCoMe-9 (NCT06611436). The…

A committee of the European Medicines Agency (EMA) has recommended that the approval of Alhemo (concizumab-mtci) be expanded to cover its use to reduce the risk of bleeding in people with severe hemophilia A and moderate or severe hemophilia B who don’t have inhibitors. The recommendation from…

People with hemophilia have significantly higher rates of depression, anxiety, stress, and overall mental distress than healthy individuals, with physical symptoms playing a role, a study in Germany found. These include “pain-related parameters, but also manifested arthropathy [joint disease] and the presence of viral infections,” the researchers wrote, which…

A single dose of Roctavian (valoctocogene roxaparvovec-rvox), a gene therapy for severe hemophilia A from BioMarin Pharmaceutical, maintains factor VIII (FVIII) levels at near-normal or normal levels for up to five years, with most patients staying off prophylaxis, or preventive treatment. These lasting benefits were observed in GENEr8-1…

Adults with hemophilia who switched from standard to extended half-life replacement treatment needed fewer infusions, had fewer bleeds per year, and adhered better to their treatment schedules, a Finnish study shows. Switching to extended half-life replacement treatment increased the mean yearly costs by nearly €19,000 (about $22,000) for…

Scientists have developed advanced liver organoids — lab-grown “mini-organs” that mimic the function of organs in the body — that are capable of growing their own functional blood vessels and producing clotting factors that could eventually be used to treat hemophilia, according to a study. In a mouse model…

The first hemophilia B patient in Germany has received the gene therapy Hemgenix (etranacogene dezaparvovec), according to CSL Behring, the therapy’s developer. “The treatment of the first patient in Germany with Hemgenix marks a milestone in hemophilia B therapy,” Christian Wieszner, managing director of CSL Behring Germany,…

Hemlibra (emicizumab-KXWH) safely reduces bleeding episodes in people with hemophilia A without inhibitors, resulting in a high rate of patient satisfaction, according to a review of published studies. “The success of [Hemlibra] underscores the potential for innovative therapeutic approaches to significantly improve patient outcomes and quality of life…

In people with hemophilia A, certain immune markers in the blood may help predict patient responses to immune tolerance induction (ITI) — a treatment regimen used for individuals who develop inhibitors after factor replacement therapy — according to a new study by researchers in Brazil. Inhibitors, neutralizing antibodies…

Preventive treatment with Hympavzi (marstacimab-hncq) can effectively reduce bleeding rates in people with hemophilia A or B who have inhibitors, or antibodies against standard replacement therapies, according to top-line data from a Phase 3 clinical trial. Hympavzi’s developer Pfizer said it’s now planning to discuss these…