New research shows that bilateral total knee arthroplasty — or reconstruction of the joint — is a safe and cost-effective treatment for patients with hemophilic arthropathy (a hemophilia degenerative joint disease). The study, titled “Simultaneous bilateral total knee arthroplasty in patients…

A major complication in scientists’ efforts to treat hemophilia A by replacing a missing or defective clotting factor is an immune response to the treatment. Researchers have discovered a connection between the body’s production of pro-inflammatory molecules known as cytokines and its immune response to clotting factor VIII therapy. They used…

Catalyst Biosciences has amended the protocol of its ongoing Phase 1/2 clinical trial investigating the highly potent recombinant human factor IX variant CB 2679d in previously treated hemophilia B patients. The amendment has shortened the study’s duration, which is a dose-escalation study to investigate the safety and drug and organism responses (pharmacokinetics and…

Shire and Rani Therapeutics have partnered to investigate the use of oral Rani Pill technology as the carrier system for clotting factor VIII in hemophilia A patients. As part of Shire’s commitment, the Irish pharmaceutical company made an equity investment in Rani. Under the agreement — whose terms weren’t…

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) 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…

Swedish Orphan Biovitrum (Sobi) announced the successful enrollment of the first hemophilia A patient in the company’s ReITIrate clinical trial. The Phase 4 study (NCT03103542), sponsored in collaboration with Bioverativ Therapeutics, is actively recruiting participants who have developed inhibitors and who have failed to respond to other therapies. Researchers will…

Researchers have found that the high price of recombinant clotting factors for patients with hemophilia B is the main cause of the economic burden of the disease in the United States. However, despite the cost, prophylaxis treatment can provide clinical benefits to patients that may reduce other costs not…

The spleen’s marginal zone (MZ) B-cells respond to blood coagulation factor VIII (FVIII) and show potential to become the target of future therapies to reduce FVIII inhibitors in hemophilia A, according to new research in mice. The study, “Marginal zone B cells are critical to factor VIII inhibitor…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status (ODD) to Catalyst Biosciences’ highly potent recombinant human factor IX variant CB 2679d (also known as ISU304) to treat hemophilia B. Catalyst is evaluating CB 2679d’s potential for subcutaneous prophylactic treatment in people with hemophilia B. The regulatory decision…