Newsroom
In our Newsroom we share some recent headlines and links to articles that may be of interest to our bleeding and clotting disorder patients, families, and providers.
Small Study Shows Extended Half-life Using Experimental Treatment for Hemophilia with Inhibitors
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.
Study Shows Hemlibra Safe, Effective in Older Patients
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.
FDA Grants Orphan Drug Status to Biopharma's B-cell Treatment for Hemophilia B
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.
Phase 3 Trial Shows Mim8 Better at Reducing Bleeds for Hemophilia A Patients than Standard Treatments
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.
New Study Shows Long Term Benefits of Gene Therapy for Severe Hemophilia A
May 15, 2024
A new study, published in the Journal of Thrombosis and Haemostasis showed significant long-term benefits of valoctocogene roxaparvovec (Roctavian) for patients with severe hemophilia A. The GENEr8-1 study was a multicenter, open-label, single-arm, phase 3 trial that aimed to show safety and efficacy data for the treatment.
FDA Approves New Hemophilia B Gene Therapy Product BEQVEZ
April 30, 2024
The FDA has approve BEQVEZ (Pfizer, Inc.) a new hemophilia B gene therapy product. It is designed with bioengineered adeno-associated virus (AAVs) vectors to introduce a working copy of the factor IX gene. This is the second hemophilia B gene therapy product to be approved in the United States.
New Long-Term Data Shows Emicizumab Safe, Effective in Hemophilia A Patients
April 16, 2024
A new long-term study showed that emicizumab (Hemlibra; Genentech) was safe and effective in hemophilia A patients without inhibitors. The analysis identified no new safety signals in patients participating in the HAVEN 3 and 4 clinical trials.
Study Shows Wristband Tracking Activity Improves Quality of Life in Severe Hemophilia Patients
April 12, 2024
A study in Spain found that severe hemophilia patients who wore activity wristbands to self-manage their physical activity saw significant improvements in their physical health and quality of life.
FDA Approves IXINITY for Use in Children 12 and Under with Hemophilia B
March 26, 2024
The US FDA approved a supplemental Biologics License for IXINITY that expands the product indication to be used for on-demand, prophylactic, and perioperative treatment of children under 12 years old. The previous indication was limited to adults and older children more than 12 years of age.
ReciBioPharm Teams up with GeneVentiv to Develop Gene Therapy for All Hemophilia Patients
March 15, 2024
ReciBioPharm and GeneVentiv Therapeutics are teaming up to advance the development of GENV-HEM, a gene therapy for all hemophilia patients, including those with inhibitors. The treatment is designed to deliver a gene coding for activated clotting factor V (FVa) an dis carried aboard adeno-associated virus 8 (AAV8, which has been modified to not cause disease in humans.
FDA Approves First Human Study of CRISPR/Cas9-based Factor 9 Gene Editing Therapy
Feb 28, 2024
A FDA has approved Intellia Therapeutics and Regeneron's plans to advance their F9 gene-editing therapy into clinical testing in humans. The collaboration includes their program focused on hemophilia B, and they are currently teamed up to advance a similar strategy for hemophilia A.
Muscle Loss Common in Hemophilia A Patients with Joint Bleeds
February 23, 2024
A study published in Haemophilia looked at how common sarcopenia (loss of muscle mass and strength) was in men diagnosed with severe hemophilia A who had recurrent joint bleeds, compared to healthy men. A modified Sonographic Thigh Adjustment Ratio was used as a measure of muscle mass.
News Content Disclaimer:
Links to articles listed may take readers to websites separate and out of the control of the CU School of Medicine and the Hemophilia and Thrombosis Center. The CU School of Medicine and the Hemophilia and Thrombosis Center are not responsible for any errors, inaccuracies or opinions that may be expressed in any of the linked articles.
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Previous articles featured on our website can be found at our archive page here. Please note that links in our archive files may take readers to websites that may have changed their link. In some cases these links do not work anymore. We periodically check and remove articles that have broken links. If you have questions about these articles, please contact us at hemophilia@ucdenver.edu.