Directly switching from Hemlibra (emicizumab) to Novo Nordisk‘s investigational Mim8 (denecimig) was well tolerated in adults and adolescents with hemophilia A, regardless of whether they had inhibitors or not. That’s according to results from the now-completed Phase 3b FRONTIER5 study (NCT05878938), which assessed the safety of switching…
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BioMarin Pharmaceutical has resubmitted a regulatory application seeking approval in Europe for its experimental gene therapy Roctavian (valoctocogene roxaparvovec) for severe hemophilia A. That resubmission, to the European Medicines Agency (EMA), included one-year follow-up data from BioMarin on the therapy’s safety and effectiveness in treating the rare genetic…
A foam roller to release tension in the fascia — a casing of fibrous tissue that surrounds and holds other tissues in place — is safe to be used by patients with knee joint disease caused by hemophilia, a Spanish has study found. When the fascia tightens around muscles,…
Despite preventive treatment, people with hemophilia A or B report lower quality of life, mainly due to poor joint health, a study in Sweden shows. No significant difference was found between these two patient groups, who report high frequency of pain, mobility problems, and mental health issues like anxiety…
ASC Therapeutics has joined forces with Charles River to scale the manufacturing of ASC618, its second-generation virus-based gene therapy for hemophilia A. The agreement expands upon their current collaboration, begun in 2019 that focused on achieving Good Manufacturing Practice (GMP)-virus manufacturing and establishing processes for adeno-associated virus (AAV) production…
A therapy designed to promote the growth of regulatory T-cells (Tregs) in the body prevented inhibitor development in a mouse model of hemophilia A that was treated with factor replacement therapy, a study showed. Inhibitors, a type of neutralizing antibody some patients develop that can make replacement therapies…
When administered at the highest dose, the investigational gene therapy SB-525 (giroctocogene fitelparvovec) prevented bleeds and the use of clotting factor VIII (FVIII) in men with severe hemophilia A, according to follow-up data from the Alta Phase 1/2 clinical trial. All five men given the highest dose…
The development of neutralizing antibodies, or inhibitors, against the clotting factor in replacement therapies administered to people with hemophilia may depend on the type of product used, a recent study has found. In previously untreated people with severe hemophilia A, or those who lack…
BioMarin Pharmaceuticals is seeking marketing approval in Europe for its investigational gene therapy, valoctocogene roxaparvovec, for the treatment of adults with severe hemophilia A. The company has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for the experimental gene therapy, formerly…
Switching to a different clotting factor VIII (FVIII) product was not linked to a greater risk of developing neutralizing antibodies against FVIII, or FVIII inhibitors, in people with hemophilia A, a recent study reported. Changing treatment also had no impact on a patient’s immune profile or on therapy efficacy.