In a real-world study in South Korea, preventive treatment with Adynovate (rurioctocog alfa pegol) was shown to be effective at preventing bleeding episodes among most children and adults with hemophilia A — findings that line up with those from clinical testing. In fact, “76.1% of patients on prophylactic…
News
Switching from standard half-life factor replacement therapies to the extended half-life treatment Elocta (efmoroctocog alfa) improves bleed control among people with hemophilia A. That’s according to an analysis of data from the real-world A-SURE study (NCT02976753), which showed that switching to Elocta, sold as Eloctate in the…
The U.S. Food and Drug Administration (FDA) has expanded its approval of Alhemo (concizumab-mtci), a daily therapy injected under the skin for preventing or reducing the frequency of bleeding episodes in people with hemophilia A or B, ages 12 and older. Sold by Novo Nordisk, Alhemo…
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,…
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