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…
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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…
Alnylam Pharmaceuticals has suspended patient dosing in all ongoing studies of its RNA therapy fitusiran to treat hemophilia A or B, after a patient died of swelling in the brain in a Phase 2 open-label extension trial. The company will work with the regulatory agencies in reviewing the cause of the…
Bayer has filled a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) requesting marketing approval for its long-acting human Factor VIII therapy, BAY94-9027, to treat hemophilia A. The therapy is designed to allow for dosing at intervals as long as once every seven days. The…
Bioverativ will collaborate with the Boston imaging company Invicro to use ultrasound imaging to improve the diagnosis and management of joint disease in hemophilia patients. “Through this innovative collaboration with Invicro, we hope to create meaningful progress in hemophilia care by helping people with hemophilia, their caregivers, physicians and treatment team monitor…
