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…
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…
The National Hemophilia Foundation (NHF) has changed its name to the National Bleeding Disorders Foundation (NBDF) to reflect its support for people with inheritable blood and bleeding disorders other than hemophilia. The new name includes a new visual identity and logo. The foundation also unveiled a new tagline…
Dosing has begun in a Phase 2b clinical trial evaluating the safety and efficacy of SerpinPC, Centessa Pharmaceuticals’ investigational therapy for hemophilia. The open-label study, called PRESent-2 (NCT05789524), is part of the company’s registrational program for hemophilia B, which includes multiple clinical trials aiming to…
Switching to a different clotting factor VIII (FVIII) product was not linked to a greater risk of developing neutralizing antibodies against FVIII, or FVIII inhibitors, in people with hemophilia A, a recent study reported. Changing treatment also had no impact on a patient’s immune profile or on therapy efficacy.
Incidences of bleeding from playing sports are rare among people with hemophilia, and more likely to occur in those with lower levels of clotting factors — proteins that play a key role in promoting blood clotting — a study in the Netherlands has found. More specifically, the study reveals…
Despite preventive treatment, people with hemophilia A or B report lower quality of life, mainly due to poor joint health, a study in Sweden shows. No significant difference was found between these two patient groups, who report high frequency of pain, mobility problems, and mental health issues like anxiety…
MRI screening can identify subclinical joint bleeding in some patients with severe hemophilia A on preventive treatment, according to a study in the Netherlands. Subclinical bleeds occur without symptoms and usually cannot be detected by standard physical and ultrasound examinations. “These MRI findings support the hypothesis that subclinical…
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended Hemlibra’s (emicizumab) approval be expanded to include people with moderate hemophilia A without inhibitors. “We’re very pleased that the CHMP’s recommendation brings us closer to potentially transforming the day-to-day lives of people in the…
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