News

A review is continuing into a resubmitted application for approval of Roctavian (valoctocogene roxaparvovec), a gene therapy for adults with severe hemophilia A, with a planned advisory committee meeting having been canceled. The U.S. Food and Drug Administration (FDA) had requested, but now no longer intends, to meet with…

The first gene therapy for adults with hemophilia B — branded as Hemgenix (etranacogene dezaparvovec) — has been approved by the U.S. Food and Drug Administration (FDA). Meant to be a one-time treatment, Hemgenix is expected to keep bleeding in check in the long term, while also allowing patients…

Despite the increasing use of preventive factor VIII (FVIII) replacement therapy, people living with hemophilia A in Japan still experience a significant disease burden, according to a recent study. Using information from health records databases, researchers found that while patients have used increasing doses and spent more on medical…

Centessa Pharmaceuticals is set to open PRESent-5, an observational feeder study of hemophilia patients who could then move in pivotal clinical trials of SerpinPC — an investigational therapy for hemophilia A and B patients, regardless of disease severity or inhibitor status — that it plans to launch…

Enzyre has raised €12 million (over $12 million) to speed the development of EnzyPad, a device that could one day allow people with hemophilia A to monitor their blood-clotting status in real time from anywhere, including their home. “We are delighted to have closed this successful financing…

The U.S. Food and Drug Administration (FDA) has scheduled its inspection of the facility in California where BioMarin Pharmaceutical would produce the gene therapy Roctavian (valoctocogene roxaparvovec) if it’s approved. The agency is reviewing a biologics license application (BLA) from BioMarin that seeks approval of Roctavian to…

Two new gene therapy vectors designed for liver diseases show promising effects in a mouse model of hemophilia B, according to a recent study. The study, “Adeno-associated virus serotype 2 capsid variants for improved liver-directed gene therapy,” was published in Hepatology. Hemophilia B is caused…

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…