News

Inspection of Roctavian Facility Set; FDA Seeks More Trial Data

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…

Heart Risk Higher in Less Active Adult Hemophilia Patients: Study

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…

Hemlibra Effective at Controlling Bleeds in Acquired Hemophilia

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…

BioMarin Resubmits Request to FDA for Roctavian’s Approval

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…