October 18, 2024
A real world study showed that eptacog beta, known as Sevenfact, was deemed safe and effective at stopping bleeds in patients with hemophilia A or B that also have inhibitors. The bypassing agent was shown to be 96% effective at resolving symptoms and controlling bleeds.
October 11, 2024
The US Food and Drug Administration approved Hympavzi (marstacimab-hncq) for routine prophylaxis to prevent or reduce frequency of bleeding episodes in adult and pediatric patients 12 and over. The treatment is designed to treat both hemophilia A and B as it targets a protein in the blood clotting process.
October 1, 2024
According to a real-world study in Japan, Hemlibra effectively prevented bleeds in patients with acquired hemophilia A. The study compared outcomes of patients with acquired hemophilia A who were treated with Hemlibra, and those who were not, finding those in the Hemlibra group had significantly less bleeding.
September 13, 2024
In a survey of doctors, researchers found that nearly two-thirds of men with moderate or severe hemophilia A have joint problems that cause pain and reduce quality of life, even when they are on factor replacement therapy to reduce bleeding. The data showed that joint problems still represent a burden in the care of these patients.
September 6, 2024
A gene editing therapy being developed by Metagenomi for hemophilia A has shown safety and durability in an ongoing pre-clinical study in three non-human primates. The results showed that after a year, two animals had normal or nearly normal factor levels. The third animal had factor levels in the mild hemophilia range.
August 20, 2024
A single dose of the gene therapy Roctavian (valoctocogene roxaparvovec-rvox) nearly zeroed out the number of bleeds and need for prophylaxis for as long as seven years. This is the longest follow-up to date for any hemophilia A gene therapy clinical trial.
August 6, 2024
A study done through the World Federation of Hemophilia shows that hemophilia has a negative effect on a person's employability. The data from the ADVANCE study showed those who received prophylactic treatments had a significantly lower probability of unemployment due to disability.
August 1, 2024
Pfizer announced positive data for their phase 3 clinical study of giroctocogene fitelparvovec, an investigational gene therapy for hemophilia A. The AFFINE study is looking at the safety and efficacy of the one time infusion and it's impact on patient's annual bleeding rate through 15 months after treatment.
July 16, 2024
A small study of TiumBio's experimental treatment, TU7710, in people with hemophilia who have inhibitors showed the half-life of the medication was five to seven times longer than a conventional hemophilia treatment for inhibitor-positive patients. With a longer half-life, the therapy should last longer and thus requiring fewer injections to stop bleeds.
July 14, 2024
The efficacy and safety of preventative treatment with Hemlibra are not affected by age or other health conditions in older people with Hemophilia A. The findings showed that outcomes for older people were consistent with those of younger people, with no significant differences in mean annualized bleed rate.
June 7, 2024
The US Food and Drug Administration (FDA) has granted orphan drug designation to BE-101, a potential treatment for hemophilia B that uses cells from the patient who receives treatment. The status provides additional benefits in the approval process for therapies meant to treat rare diseases.
May 22, 2024
Results from a Phase 3 FRONTIER 2 (NCT05053139) study showed that Novo Nordisk's investigational antibody therapy was seen to be better than on-demand or standard preventative treatments at reducing bleeding episodes in patients with hemophilia A, regardless of inhibitor status.
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