The full range of hemophilia treatments should be made available to patients without consideration of cost or other barriers, leaders of patient associations and others argued at a public meeting hosted by the Institute for Clinical and Economic Review (ICER) to consider its recent analysis of hemophilia A therapies. The…
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Weekly treatment with the experimental therapy marstacimab (PF-06741086) was found to significantly reduce bleed rates for adult and adolescent hemophilia A and B patients without inhibitors relative to standard therapies. In so doing, it met the main goal of the pivotal Phase 3 BASIS clinical trial, designed to…
Giroctocogene fitelparvovec, formerly known as SB-525 or PF-07055480, is a gene therapy being developed to reduce the risk of bleeding episodes in people with hemophilia A.
Advate (antihemophilic factor, octocog alfa) is a recombinant (lab-made) clotting factor VIII (FVIII) concentrate used to prevent and control bleeding in children and adults with hemophilia A.
Xenetic Biociences has received a $3 million milestone payment from its partner Shire for advancing the Phase 1/2 clinical trial of PSA-recombinant SHP656, a factor VIII protein, evaluating the drug’s effectiveness as a long-acting therapy for hemophilia A. Shire’s main objective behind the agreement is to develop a novel FVIII…
Novo Nordisk has resumed its Phase 3 clinical trials investigating concizumab as a potential treatment for hemophilia A and B, the company announced in a press release. The trials were paused in March due to the occurrence of non-fatal thrombotic (blood-clotting) events in three patients. In collaboration…
Adults and adolescents with hemophilia A or hemophilia B without inhibitors experienced significantly fewer bleeding episodes when using concizumab as a prophylactic (preventive) treatment compared with those who weren’t on any prophylaxis. That’s according to data from explorer8 (NCT04082429), an ongoing Phase 3 clinical study in which…
Recombinate (antihemophilic factor recombinant) was a recombinant (lab-made) clotting factor VIII (FVIII) concentrate that was approved to prevent and control bleeds, including those occurring during surgery, in children and adults with hemophilia A. It was administered via intravenous, or into-the-vein, injection.
Half of the children in Chile with severe hemophilia A and B exhibit joint damage despite receiving preventive treatment with clotting factors, a study reports. Damage to the cartilage is the main sign of joint impairment in children older than 8 years, while synovitis, or inflammation of the synovial…
The rate of intracranial hemorrhage (ICH) — a bleed in the brain — is higher in children and adults with hemophilia than in the general population, an Italian study suggests. ICH rates were particularly high among adults with mild disease and with high blood pressure. Most patients received only…