The other day, I picked up “MacDonald the Younger” from hemophilia camp. I enjoyed the trip through the mountains. The air felt fresh as I grew anxious to see my son. I wanted to hear all about his camp experience. What friends did he see? Did he try the…
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The approval by the U.S. Food and Drug Administration (FDA) of BioMarin Pharmaceutical’s Roctavian (valoctocogene roxaparvovec-rvox) late last month means that eligible adults with severe hemophilia A will soon have access to a gene therapy for the first time. “Adults with severe hemophilia A live with several limitations,…
Delaying the start of immunosuppressive treatment in adults diagnosed with acquired hemophilia A who are receiving Hemlibra (emicizumab-kxwh) to prevent bleeding may reduce the risk of infections and extend survival, a clinical study found. Patients in the Phase 2 study (NCT04188639 received Hemlibra for 12 weeks while immunosuppression…
The U.S. Food and Drug Administration (FDA) has accepted a resubmitted biologics license application (BLA) from BioMarin Pharmaceutical requesting approval of the gene therapy Roctavian (valoctocogene roxaparvovec) to treat adults with severe hemophilia A. The FDA is now expected to decide by the end of March 2023 on whether…
Hemophilia is a bleeding disorder caused by a mutation in genes that provide instructions for blood clotting factors — the proteins that help in blood clotting. Mutations in the genes for clotting factors VIII, IX, and XI result in their deficiency, causing hemophilia A, hemophilia B, and hemophilia C, respectively.
A combination of Cyklokapron and Feiba — usually avoided due to a perceived risk of blood clots — appears to be safe and effective for treating patients with acquired hemophilia A (AHA), though these early study results need to be confirmed in clinical trials, researchers in Italy say. The study, “…
The investigational gene therapy SB-525 is tolerated well by patients, and dose-dependent responses are positive, according to preliminary results of a Phase 1/2 clinical trial of hemophilia A. The potential gene therapy is being developed as a partnership between Sangamo Therapeutics and Pfizer. It consists of…
Two clinical trials launching in December will use Octapharma USA’s Wilate, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection, in patients with hemophilia A. Hemophilia A is an X-linked hereditary bleeding disorder that results from either reduced or absent levels of…
Prophylactic hemophilia treatment can save patients with severe forms of the disease from the long-term consequences of joint damage and other complications. A report by Dr. Suchitra S. Acharya, MD, of Hofstra Northwell School of Medicine in New York, gives a detailed overview of the current state and future perspectives…
Oxidation of clotting protein factor VIII (FVIII) increased the immune response to the replacement therapy in a mouse model of severe hemophilia A, a study found. Conducted by researchers in France, the study, “Oxidation of factor VIII increases its immunogenicity in mice with severe hemophilia…