News

Physical function assessment can be a good predictor of lower bone mineral density and osteoporosis development associated with hemophilia, according to a report published in the American Journal of Blood Research. Therapies to treat hemophilia made available in the past decades have significantly improved patients’ outcomes. This extended survival…

The U.S. Food and Drug Administration (FDA) has approved an update to the label of Alprolix, based on long-term efficacy and safety data from two Phase 3 studies, the B-YOND extension trial and the Kids B-LONG pediatric study, manufacturer Bioverativ announced. This label extension, which adds pediatric data, further supports…

Nuwiq, a fourth-generation genetically engineered version of blood-clotting factor VIII, is a safe and effective way of controlling bleeding in people with severe hemophilia A during and after surgery, a study reports. The research, “Efficacy and safety of Nuwiq® (human-cl rhFVIII) in patients with severe haemophilia A undergoing…

Administration of once-weekly Hemlibra (emicizumab-kxwh) significantly reduced bleeding rates in patients with Hemophilia A compared to other clotting factor VIII prophylaxis (preventive) therapy, according to the latest results of the Phase 3 HAVEN 3 study announced by Roche. Developed by Chugai Pharmaceutical, Roche, and Genentech,…

Officials of the U.S. Food and Drug Administration and Alnylam Pharmaceuticals have agreed on safety measures and a “risk mitigation strategy” to allow the resumption of clinical studies of fitusiran, the company said in a recent press release. All ongoing trials of fitusiran had been suspended after the Sept. 7 death…

The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to rofecoxib (TRM-201), an non-opioid investigational pain treatment for hemophilic arthropathy (HA, a hemophilia degenerative joint disease) under development by Tremeau Pharmaceuticals. Rofecoxib is a COX-2 selective non-steroidal anti-inflammatory drug (NSAID) with a well-established efficacy profile. It is…

The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform package. A similar package before the Senate Finance Committee does not repeal the credit…

The European Medicines Agency has recommended marketing authorization for Shire’s Adynovi (BAX-855) as a treatment for adults and adolescents with hemophilia A. Approval would mean the therapy could be used twice a week to prevent bleeding episodes and to counter bleeding when episodes do occur. The U.S. Food and Drug Administration…

The U.S. Food and Drug Administration (FDA) has approved Hemlibra (emicizumab-kxwh) for routine prophylaxis to prevent or lessen bleeding episodes in children and adults with hemophilia A with factor VIII inhibitors, Genentech announced. Hemlibra is first newly approved treatment for this patient group in almost 20 years, the…

An ongoing Phase 1/2 clinical trial evaluating a gene therapy, SPK-9001, in people with hemophilia B is expanding its enrollment by up to five more patients, who will be given a new version of the therapy, its developer announced. One patient in this group has already received SPK-9001 produced under…