The European Medicines Agency (EMA) is asking for a full year’s worth of data from a Phase 3 trial of Roctavian as it considers approving this potential gene therapy for adults with severe hemophilia A, its developer, BioMarin, reported. The request by the regulatory agency for the European…

Clinical data from the ongoing Phase 2/3 trial data evaluating MarzAA (marzeptacog alfa activated) in patients with Hemophilia A or B with inhibitors continues to demonstrate the treatment’s potential to prevent bleedings. The most recent trial results were discussed at the 12th Annual Congress of the European Association for Haemophilia…

BioMarin is considering pricing its hemophilia A gene therapy Valrox (valoctocogene roxaparvovec), should it be approved, at $2 million to $3 million. That range would make Valrox the world’s most expensive one-time therapy. But, the company argues, this first gene therapy for any form of inherited hemophilia could…

Healthcare costs, particularly related to disease-specific medications, are especially high for people with hemophilia A or hemophilia B on preventive treatments and/or for those with inhibitors. That’s according to a study in France, which also found that patients have a significant clinical burden, with a higher risk of…

Shire recently announced that the U.S. Food and Drug Administration (FDA) has approved its Baxject III reconstitution system for Adynovate, an approved treatment for Hemophilia A. This system will allow patients and caregivers to prepare the therapy with fewer steps, minimizing the chances for possible errors. Adynovate is a recombinant Factor VIII (rFVIII) treatment with…

As a long-term preventive treatment, fitusiran leads to sustained reductions in bleed frequency in people with moderate-to-severe hemophilia A or B regardless of inhibitor status, early results from an extension study show. The findings were presented at the recent World Federation of Hemophilia Virtual Summit and…

Some $80.3 million in financing will support the first clinical trial of Sigilon Therapeutics’ new hemophilia A cell therapy candidate, SIG-001, the company said. The trial is expected to begin by June. Last August, the U.S. Food and Drug Administration (FDA) granted the potential cell therapy…

Japan’s Ministry of Health, Labor and Welfare has extended Hemlibra (emicizumab)’s approval to include routine prophylaxis to prevent or lower the frequency of bleeds in people with acquired hemophilia A. Its decision comes seven months after Hemlibra’s maker, Chugai Pharmaceutical, applied for extended use. Hemlibra was previously approved in…

MGX-001, a gene-editing therapy being developed by Metagenomi for hemophilia A, has shown promising safety and durability in an ongoing preclinical study in three nonhuman primates, the company has announced. Results showed that after one year, two animals had normal (82%) or nearly normal activity levels (41%) of…

Using a baby’s own umbilical cells as vehicles to deliver factor VIII (FVIII) — the missing or defective clotting protein in hemophilia A — may be an effective, long-lasting, and more affordable therapeutic approach for this blood disorder, an early study suggests. The findings “lay the groundwork for future…