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.
Survey Shows Little Interest in Gene Therapy for Many Hemophilia Patients
March 20, 2026
A small survey in Germany found that many people with severe hemophilia are not interested in receiving gene therapy. Researchers found that despite having only a general knowledge of the treatment option, more than half of those surveyed had a predominantly rejective attitude toward the therapy.
CSL Behring Announces Temporary Disruption in Hemgenix Supply
March 18, 2026
CSL Behring announced that its hemophilia B gene therapy, Hemgenix, was experiencing a temporary global stockout of the treatment. The company shared that the supply issue was not related to safety or effectiveness of the medication, but reflects the complexity of manufacturing gene therapies while maintaining regulatory and quality standards.
Study Shows Lower-Dose Gene Therapy Effective in Hemophilia A
March 17, 2026
A Phase 1 pilot study in China showed their experimental gene therapy GS001 safely increased the levels of factor VIII for hemophilia A patients for almost three years. The treatment reduced bleeds and the need for replacement therapy.
Gene Therapy for Hemophilia B Continues to Show Factor Production after Six Years
March 10, 2026
A single infusion of Beqvez, a now-discontinued gene therapy by Pfizer for hemophilia B, led to sustained production of factor IX for up to six years. The treatment reduced bleeding episodes and eliminated the need for routine preventative infusions in most patients. The study was published in Blood Advances.
BioMarin Pulls Gene Therapy Roctavian from US Market
February 26, 2026
BioMarin Pharmaceuticals has announced it is pulling the hemophilia A gene therapy Roctavian from the US Market. The move followed an announcement in October 2025 that it was looking for a buyer for the expensive therapy. The treatment will remain available to the US community through the end of May 2026.
Hemophilia Patients Face 46% Higher Risk of Bone Fractures
January 30, 2026
A large-scale Canadian study showed that people living with hemophilia face a 46% higher risk of bone fractures than the general population. The risk increases with severity and for those who have inhibitors.
NBDF and WFH Issue Statement on Severe Adverse Event with Hympavzi
December 22, 2025
The National Bleeding Disorders Foundation (NBDF) joined the World Federation of Hemophilia (WFH) in issuing a joint statement regarding a severe adverse event with Hympavzi. An individual on the prophylactic treatment suffered a fatal thrombotic stroke after a minor surgery. The editors note indicates that the circumstances are multi-factorial, including co-existing conditions.
Extended Dosing Interval of Emicizumab May Offer Comparable Protection for Hemophilia A Patients
December 2, 2025
Many people with hemophilia A may be able to use significantly less emicizumab while maintaining similar protection against bleeding. This is according to interim results from the ongoing DosEmi trail that followed patients that extended their dosing interval, typically from 7 to 14 days without increasing doses.
Study Shows Hemlibra Safely Supports Joint Health in Children with Hemophilia A
November 21, 2025
An analysis in Japan showed that Hemlibra supports joint health in children with hemophilia A without inhibitors over three years of treatment. The ongoing study is well tolerated and appears to maintain or improve joint health in children under the age of 11.
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.
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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