Newsroom

FDA Grants Fast Track Status to 1-Time Stem Cell Therapy for Hemophilia A

May 5, 2026

The FDA has awarded both fast track and rare pediatric disease designations to a single-use stem cell therapy being developed for hemophilia A by Expression Therapeutics. The decision follows a Phase 1 clinical trial showing a single administration of the therapy led to sustained factor expression and the annual bleeding rate dropped to zero.

New Study Finds Bispecific Antibody Prophylaxis Reduces Bleeding in Hemophilia A

May 4, 2026

Researchers in Italy have found that prophylaxis with Mim8, a novel bispecific antibody designed to replicate function of activated factor VIII, was superior to both on-demand therapy and clotting factor concentrate prophylaxis in reducing annualized rate of bleeding events, regardless of inhibitor status. 

Research Shows Regular Excercise Eases Chronic Pain in Hemophilia

April 17, 2026

A study published in Pain Management Nursing reviewed published research about the effect of physical excercise, including aerobic, strength training, and balance exercises, found that regular exercise may help ease chronic pain for those with hemophilia.

Study Finds Protein Marker May Detect Early Joint Damage for Hemophilia A Patients

April 3, 2026

A study in Greece compared the YKL-40 protein levels - a protein associated with inflammation - in a small group of boys and men with severe hemophilia A and found that those with known joint damage had signficantly higher levels. The findings offer a potential new way to monitor joint damage in bleeding disorders patients.

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.