News

U.S. hemophiliacs who use patient-reported outcome (PRO) instruments can provide reliable information on their disease burden, finds a new study that appeared in the journal Patient Preference and Adherence. In the study, “Reliability of patient-reported outcome instruments in US adults with hemophilia: the Pain, Functional Impairment and Quality…

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…

Three U.S. bleeding disorder patient organizations recently received funding support for their patients’ rights advocacy campaigns. The Milwaukee, Wisconsin, headquartered Great Lakes Hemophilia Foundation (GLHF), the New York City Hemophilia Chapter (NYCHC), and the Columbus, Ohio-based Ohio Bleeding Disorders Council (OBDC) have been…

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…

Catalyst Biosciences recently announced that its collaborator, ISU Abixs, has completed dosing of the first subcutaneous cohort in an ongoing Phase 1/2 proof-of-concept clinical trial in people with severe hemophilia B. This patient group is the second of up to five involved in the study. CB 2679d (also known as ISU304)…

Researchers have found that adults with severe hemophilia A coupled with pre-existing joint disease benefit most from prophylaxis to improve function, quality of life, and activities, and to better manage pain. The study, “Effect of late prophylaxis in hemophilia on joint status: a randomized trial,” was published in…

The Boston-based Institute for Clinical and Economic Review (ICER) says a “draft scoping document” outlining its planned comparative review of emicizumab‘s clinical effectiveness and value in treating hemophilia A will be available until Sept. 29. ICER’s report will be subject to a public deliberation in March 2018 by the…

Bioverativ and Bicycle Therapeutics will jointly discover, develop and market new therapies to treat hemophilia and sickle cell disease, the two companies announced. These future therapies will be based on Bicycle’s proprietary peptide development platform. “We believe our Bicycle platform has extremely broad therapeutic potential and we are excited to work…

Results of the first group of patients with hemophilia B treated with Catalyst Biosciences’ lead candidate CB 2679d in a Phase 1/2 clinical trial demonstrated its improved stability and activity compared to Pfizer’s BeneFix standard-of-care replacement therapy. CB 2679d (also known as ISU304), was designed to be the…

Dimension Therapeutics may soon become a wholly owned subsidiary of Regenxbio, following the terms of an agreement between the two companies. The merger agreement, if finalized, will add Dimension’s investigational drug candidates being developed to Regenxbio’s pipeline, including DTX201 for the treatment of hemophilia A. DTX201 was being developed…