The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Baudax Bio’s investigational regulatory T-cell therapy TI-168 for the treatment of hemophilia A with inhibitors. Orphan drug status is given to therapies intended to treat rare conditions, defined as those affecting less than 200,000 people in…
Altuviiio (efanesoctocog alfa) has been approved in Japan and Taiwan as a treatment to prevent and control bleeding in people with hemophilia A. The Japanese Ministry of Health, Labor, and Welfare (MHLW) granted Altuviiio marketing authorization on Sept. 25, according to Sanofi, the company that markets…
Treatment with Idelvion (albutrepenonacog alfa) was effective in a real-world setting at controlling bleeds for five people with hemophilia B at a center in Spain. That’s according to data reported in the study, “Benefits of rIX-FP prophylaxis in patients with Haemophilia B: real-world evidence from…
The U.S. Food and Drug Administration (FDA) has cleared for laboratory use CRYOcheck Factor VIII Deficient Plasma with VWF, a way of detecting factor VIII (FVIII) deficiency in blood samples. Now available in the U.S., Precision Biologic’s manufactured plasma — the liquid part of blood that…
About a quarter (24.4%) of men with hemophilia A and B seen at a clinic in Turkey met the clinical threshold for depression, and nearly three-quarters (74.4%) had anxiety, according to a recent analysis. Having a high school degree was linked to the presence of depression, whereas having…
The U.S. Food and Drug Administration (FDA) is considering updating the prescribing information for Altuviiio (efanesoctocog alfa) to include final data on a clinical trial that tested the now-approved therapy in children with hemophilia A. Sanofi, which markets Altuviiio in the U.S., submitted a supplemental biologics license…
To help advance new treatments for hemophilia and other blood disorders, Pathway to Cures (P2C) has invested $250,000 in Anvesana, a company working to develop therapies based on RNA biology. “Anvesana is delighted to be working with Pathway to Cures to develop therapeutics that will bring life altering…
Switching from on-demand treatment to regular preventive therapy with extended half-life products may mean fewer bleeds, healthier joints, and better quality of life for people with hemophilia. That’s according to a new analysis of Phase 3 study data from trials involving more than 100 hemophilia patients that spanned a…
A person in Germany with severe hemophilia A has been treated with Roctavian (valoctocogene roxaparvovec-rvox), the first time the gene therapy has been used commercially in Europe, according to BioMarin Pharmaceutical, its developer. “Today represents an important milestone for the hemophilia community and for patients and…
The protein missing or defective in people with hemophilia A, called blood coagulation factor VIII (FVIII), is able on its own to stimulate immune responses in a mouse model, a study suggests. This immune response to FVIII is dependent on a set of distinct immune cells within the spleen,…
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