Adults with moderate-to-severe hemophilia engage in less physical activity and have reduced physical fitness relative to healthy adults, according to a recent study. Data also indicated these patients had more indicators of abdominal fat accumulation and more often had high blood pressure than healthy people, which could overall reflect…
Health Canada has expanded its approval of Rebinyn (nonacog beta pegol) as a routine preventive treatment for bleeding episodes in hemophilia B to patients under age 18. The therapy, developed and marketed by Novo Nordisk, is now available to all hemophilia B patients as a preventive (prophylactic)…
Patients with moderate to severe hemophilia A who switched to Kovaltry (octocog alfa) showed no worsening of their annual bleeding rate, according to real-world data from a Phase 4 study. Adhering to the therapy remained high and patients reported high treatment satisfaction. Overall, the study “confirms and extends clinical trial…
One-month treatment with Hemlibra (emicizumab) and an immunosuppressive medication were effective for patients with acquired hemophilia at rapidly controlling bleeding and reducing the use of other therapies, a single-center study shows. The researchers also noted that using less toxic immunosuppressive agents such as rituximab was effective at blocking…
The National Institutes of Health (NIH) is supporting a university’s research into the risk factors and underlying mechanisms involved in the development of neutralizing antibodies, or inhibitors, against replacement therapies in people with hemophilia A. The $6.6 million, three-year grant from the NIH’s National Heart, Lung, and Blood Institute…
The U.S. Food and Drug Administration (FDA) has accepted a resubmitted biologics license application (BLA) from BioMarin Pharmaceutical requesting approval of the gene therapy Roctavian (valoctocogene roxaparvovec) to treat adults with severe hemophilia A. The FDA is now expected to decide by the end of March 2023 on whether…
The U.S. Food and Drug Administration (FDA) has cleared the biotechnology company TeraImmune to launch a Phase 1/2a clinical trial testing TI-168, its regulatory T-cell therapy designed to eliminate inhibitors in people with hemophilia A. The upcoming trial is expected to enroll up to 18 people with congenital…
BioMarin Pharmaceutical has resubmitted an application to the U.S. Food and Drug Administration (FDA) seeking approval of Roctavian (valoctocogene roxaparvovec), potentially the first gene therapy for adults with severe hemophilia A. The FDA delayed the company’s initial approval request in 2019, with the agency requiring two more…
COVID-19 vaccines, because they work to stimulate the immune system, may be an emerging cause of autoimmune conditions like acquired hemophilia A, according to a case report. “This report aims to highlight the risk of … [acquired hemophilia A] following an immune stimulus, thus improving our knowledge regarding possible…
A significant proportion of bleeds in people with hemophilia A are left untreated, according to an analysis of data from an observational study. Because only treated bleeds are typically reported in clinical trials, these findings suggested the full disease burden is not adequately captured in many studies. As such,…
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