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…
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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…
Directly switching from Hemlibra (emicizumab) to Novo Nordisk‘s investigational Mim8 (denecimig) was well tolerated in adults and adolescents with hemophilia A, regardless of whether they had inhibitors or not. That’s according to results from the now-completed Phase 3b FRONTIER5 study (NCT05878938), which assessed the safety of switching…
People with hemophilia A in China see major health challenges, such as frequent joint bleeding, joint malformation, and high rates of hospital visits, primarily owing to the limited use of preventive therapies, a real-world study shows. After a bleeding episode, most patients rely on on-demand treatment, which is less…
The benefits of a one-time gene therapy were sustained for up to 13 years among men with severe hemophilia B with no new safety concerns emerging, according to newly published results from a clinical trial. The findings, which encompass the longest ever follow-up for a gene therapy study in…
