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.

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.


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. 


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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