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.
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.
Trial Shows Roctavian, Gene Therapy, Maintains Effectiveness after Seven Years
February 7, 2024
Over seven years of follow-up, a single dose of Roctavian (valoctocogene roxaparvovec-rvox) reduced the yearly bleeding rate in adults with severe hemophilia A by up to 96%. According to an update on a Phase 1/2 clinical trail in the UK that is testing the safety and efficacy of the gene therapy at various doses, no new safety issues were found.
NBDF Names Phil Gattone, M.Ed as Next CEO
January 31, 2024
The National Bleeding Disorders Foundation (NBDF) Board of Directors announced that Phil Gattone, M.Ed will be their next president and chief executive officer. Gattone was president and CEO of the Epilepsy Foundation of America from 2012-2019 and is succeeding Len Valentino, MD, who was president and CEO of the NBDF since 2020.
Jivi Safe, Effective for Hemophilia A in Long-term Study
January 17, 2024
After almost three years of treatment with Jivi (damoctocog alfa pegol), a multinational group of hemophilia A patients showed a reduced annual bleeding rate. A study, looking at real world effectiveness of the treatment, showed there were no reported side effects or discontinuations.
First US Patient Treated with Hemophilia A Gene Therapy Outside a Clinical Trial
January 12, 2024
The first hemophilia A gene therapy infusion to be delivered outside a clinical trial setting was recently given to a patient in California. The patient, who has severe hemophilia A, received a single infusion of Roctavian (BioMarin) at CIBD in Orange, CA.
Three Year Study Shows SerpinPC is Safe and Reduces Bleeds in Hemophilia A & B
December 22, 2023
In a phase 1/2a clinical trial by Centessa Therapeutics, SerpinPC, a subcutaneous injection given every two weeks, showed the treatment to be safe and that bleeds of patients were reduced to almost zero in adults with hemophilia A or B. The patients in the study were in their third year of treatment and had their annualized bleeding rate drop by 96%.
Hemgenix Lowers Bleeding Rates and the Rate of Replacement Therapy
Dec 20, 2023
Data from the Phase 3 HOPE-B trial shared at the 65th American Society of Hematology (ASH) Annual meeting shows bleeding rates and the use of replacement therapies among men with severe or moderately severe hemophilia B remain low at least three years after a single dose of the approved gene therapy Hemgenix (etranacogene dezaparvovec).
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.
Looking for an older article?
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.