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.
Takeda Will Discontinue Two Treatments for Hemophilia
March 18, 2025
Takeda Pharmaceuticals announced they will discontinue two of its hemophilia treatments: HEMOFIL-M and RECOMBINATE. They shared other factor VIII products as alternative medications-contact your pharmacy if you have questions about this change.
Study: Children with Hemophilia Have Problems with Dynamic Balance and Falls
March 18, 2025
In a study published in the journal Gait & Posture, children with hemophilia were found to have more problems with dynamic balance (the ability to maintain balance while moving) and had an increased risk of falls compared to their healthy peers.
Pfizer Discontinues Hemophilia B Gene Therapy Beqvez
February 25, 2025
Pfizer has announced they are ceasing global development of Beqvez, their hemophilia B gene therapy product. The treatment was approved by the FDA in Spring of 2024.
CSL Reports on Four Years of Data for Gene Therapy Hemgenix
February 9, 2025
CSL shared four years of results from its pivotal HOPE-B study that demonstrates the long-term durability and safety of etranacogene dezaparvove-drlb (Hemgenix), the first FDA approved gene therapy for hemophilia B. Findings showed 94% of patients treated with a one-time infusion of Hemgenix were able to maintain near normal factor levels.
FDA Approves New Non-Opioid Treatment for Pain
January 31, 2025
The US Food and Drug Administration has approved suzetrigine (Journavx) 50 mg oral tablets, a first-in-class non-opioid analgesic developed to treat moderate to severe acute pain in adults. It works by targeting a pain-signaling pathway involving sodium channels in the peripheral nervous system, before pain signals reach the brain.
FDA Approves First Subcutaneous Prophylaxis for Hemophilia With Inhibitors
January 29, 2025
The FDA has approved Concizumab-mtci, known as Alhemo, the first subcutaneous therapy for the prophylactic treatment of both hemophilia A or B in adults and children ages 12 and older. The treatment offers a much needed alternative for hemophilia B patients, and adds an option for those with hemophilia A with inhibitors.
Be Biopharma Secures Financing to Advance Hemophilia B Treatment BE-101
January 17, 2025
Be Biopharma has raised $92 million in series C financing to support the first in-human Phase 1/2 trial of BE-101, the company's B-cell therapy candidate for hemophilia B. The treatment is designed to use a patient's own immune B-cells, genetically modify them, and infuse them back into the patient.
FDA Grants Fast Track Designation to Star Therapeutics' VWD Therapy
January 10, 2025
The US FDA has granted Fast Track designation to the investigational von Willebrand disease (VWD) therapy VGA039. The therapy is a subcutaneously-delivered monoclonal antibody therapy designed to target Protein S as a means of restoring proper blood clotting in people with all types of VWD.
Phase 3 Gene Therapy Trial Shows Giroctocogene Fitelparvovec Safe, Effective
December 13, 2024
Nearly two-thirds of hemophilia A patients given a single infusion of the investigational gene therapy giroctocogene fitelparvovec were free of bleeding episodes almost three years after treatment. The data from the phase 3 AFFINE clinical trial was shared at a presentation given at the American Society of Hematology (ASH) annual meeting.
Investigational Hemophilia A Gene Therapy Updates Shared at ASH
December 13, 2024
Expression Therapeutics announced phase 1 clinical trial results based on ET3, the company's investigational gene therapy. It is a therapy developed with novel lentiviral vectors. The therapy uses repurposed retroviruses to introduce factor VIII transgene to deliver targeted genetic material.
Potential Hemophilia Treatment SerpinPC Halted by Centessa
November 15, 2024
Centessa Pharmaceuticals has discontinued their clinical development of their experimental under-the-skin therapy SerpinPC. The therapy was found safe and well tolerated in a recent trial, but the company decided to focus their investments on other areas after Pfizer's Hympavzi, which used a similar mechanism, was approved by the FDA.
Study Finds Missing Clotting Proteins in Hemophilia Patients Can Directly Affect Bone Health
November 8, 2024
A recent study from Italy shows new findings on why bone problems are a common issue among hemophilia patients. The study looked at missing clotting proteins and compared experiments on cells gathered from healthy donors and hemophilia patients.
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.