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.
Biomarin Plans to Sell Rights to Roctavian, FDA Approved Hemophilia A Gene Therapy
November 7, 2025
The recently approved gene therapy for hemophilia A, Roctavian, is potentially set to be licensed to another company which will take over the commercialization. The gene therapy has shown to drastically reduce bleeds for hemophilia A patients, but the therapy has a substantial price tag of nearly $3 million in the US making it hard to find insurers to cover it.
Trial Shows Once-Weekly Marstacimab Reduces Bleeding Rates for Hemophilia A and B Patients
November 4, 2025
According to a study recently published in Blood, Marstacimab, a monoclonal antibody approved for prophylactic treatment of hemophilia A and B patients, bleeding rates were reduced significantly and the treatment was well tolerated.
Star Therapeutics Gains New Financing to Boost Investigational von Willebrand Disease Therapy
October 2, 2025
Star Therapeutics announced that they recently acquired new financing totaling $125 million that will help their company continue to develop their investigational von Willebrand disease therapy VGA039. The FDA fast tracked the subcutaneous monoclonal antibody therapy in January 2025 and the treatment is in a phase 3 trial in the US.
Study Finds Infection is Most Common Cause of Death in Acquired Hemophilia Patients
September 5, 2025
A recent registry study in Spain showed that infection and related complications pose a serious risk to those with acquired hemophilia A. The study showed that of those who had AHA (a form of acquired hemophilia A), about one quarter of the patients died, and of those, infection was the most common cause of death, accounting for 51.6% of cases.
Study Shows Spinal Stenosis May Be a Complication in Older Hemophilia Patients
September 2, 2025
A study out of Ireland showed that spinal stenosis, where there is a narrowing of the spaces in the spinal canal, may be a complication in aging people with hemophilia. The study showed that increased awareness may make a difference in treating patients as they age.
Discovery of F8 Mutation May Lead to Better Bleeding Control for Hemophilia A Patients
August 22, 2025
A team of scientists at the University of Colorado Anschutz Medical Campus discovered a F8 genetic mutation that caused recurrent blood clots in a young man since birth. The naturally occurring mutation could lead to new insights on treatments for those whose blood does clot correctly, such as those with hemophilia.
FDA Approves Alhemo for Hemophilia Patients without Inhibitors
August 14, 2025
The FDA has approved concizumab-mtci, known as Alhemo, for use in the treatment of both hemophilia A or B in adults and children ages 12 and older with or without inhibitors. This is an expansion of previously the approved treatment for those with inhibitors after a trial showed reduction of bleeding rates for those without inhibitors.
Study Shows Higher Rates of Depression, Anxiety for Hemophilia Patients
July 25, 2025
A study done in Germany shows that hemophilia patients have a significantly higher rate of depression, anxiety and other mental distress than healthy individuals, with physical symptoms playing a role.
Study Indicates Immune Markers May Predict ITI Response in Hemophilia A Patients
July 1, 2025
According to a recent study, markers in the blood may help predict patient responses to immune tolerance (ITI), a treatment used for those who develop inhibitors to factor replacement therapy. ITI is an intensive treatment aimed at eliminating inhibitors, but the study found that those who failed ITI had a higher concentration of certain markers compared with those who completed the treatment.
Trial Shows Hympavzi Lowers Bleeding Rates for Hemophilia Patients with Inhibitors
June 27, 2025
Results from a Phase 3 clinical trail show that Hympavzi (marstacimab-hncq) a subcutaneous, once-a-week injection, can reduce bleeding rates in people with hemophilia A or B who have inhibitors. Pfizer, the developer, is working to get Hympavzi approved for prophylactic therapy for patients with inhibitors.
Study Shows Gene Therapy Benefits for Hemophilia B Last More than a Decade
June 17, 2025
In the longest ever follow-up yet for a gene therapy study, recent findings showed that patients who received FDA approved AAV gene therapy (etranacogene dezaparvovec) for hemophilia B (Hemgenix) over 13 years ago still have sustained benefits with no new safety concerns emerging. Since being administered the gene therapy, FIX levels remained stable and were highest in those given the highest dose.
FDA Approves Jivi for use in Children with Hemophilia A as Young as 7 Years-old
May 20, 2025
The Food and Drug Administration has approved Jivi (damoctocog alfa pegol) that now extends to children as young as 7 years old. Jivi is an extended half-life replacement therapy, so it lasts longer in the body than traditional treatments. It is approved for this age group for both on-demand and prophylactic treatment of hemophilia A.
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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