CSL Behring, the biopharmaceutical company behind the hemophilia B gene therapy HEMGENIX ® (etranacogene dezaparvovec-drlb), provided an update to the bleeding disorders community regarding the availability of the treatment. In a letter to the hemophilia B community, the company stated that they have “reestablished limited supply in April 2026,” and that they remain committed to actively managing the existing supply.

In a letter sent to healthcare providers, Ferring Pharmaceuticals announced that STIMATE® (desmopressin acetate) nasal spray is expected to be available in the United States beginning October 2026.

Federal:

Medicaid Work Reporting Requirements

The U.S. Food and Drug Administration (FDA) has approved an expanded indication for the hemophilia treatment HYMPAVZI® (marstacimab-hncq) to now include people with hemophilia A or B.

Iron deficiency, with and without anemia, is the most common micronutrient deficiency around the world. People with bleeding disorders have a higher risk of developing these conditions due to blood loss from bleeding episodes. Until now, there were no standardized guidelines for screening for iron deficiency in people with bleeding disorders. That has changed with the release of NBDF’s Medical and Scientific Advisory Council’s (MASAC) new recommendations that give doctors and other clinicians clear guidance on how to address this issue. 

On June 1st, 2026, the federal Centers for Medicare and Medicaid Services (CMS) released its long-awaited rule providing guidance to state Medicaid programs on how to implement the Medicaid work reporting requirements and eligibility limits enacted last year as part of HR 1, the One Big Beautiful Bill Act. 

On May 22nd, Representatives Julie Johnson (D-TX-32), Joe Wilson (R-SC-02), and Rep. Sarah McBride (D-DE-At Large) introduced the Fostering Effective Diagnosis and Treatment for Underserved Populations with Bleeding Disorders Act (FED UP with Bleeding Disorders Act, H.R. 8794), federal legislation introduced in the United States Congress aimed at improving diagnosis, treatment, and research equity.

Jacob Murdock, current senior executive director of the Nevada Chapter of the National Bleeding Disorders Foundation (NBDF Nevada), will also assume leadership of the organization’s Nebraska chapter, NBDF Nebraska.

The Idaho Chapter of the National Bleeding Disorders Foundation (NBDF Idaho) and the Snake River Hemophilia & Bleeding Disorders Association (Snake River) have announced plans to merge, forming a single statewide organization that will operate as NBDF Idaho within the National Bleeding Disorders Foundation’s national chapter network.

The National Bleeding Disorders Foundation (NBDF) is proud to announce that Maria E. Santaella, PhD(c), MSN, RN-BC, CPHON, senior vice president of research strategy, has been named editor in chief of Bleeding Disorders Care & Practice.

The National Bleeding Disorders Foundation, a leading U.S. organization dedicated to improving care for people with bleeding disorders, has announced its 2025 NBDF-Takeda Clinical Fellowship Awardees: Deeksha Katoch, MBBS, and Peter Zhao, MD.

Federal: 

Strong Support for FY27 Funding for Federal Bleeding Disorders Programs 

The National Bleeding Disorders Foundation (NBDF) is alerting the community to updated information from Takeda Pharmaceutical Company regarding the discontinuation timelines for HEMOFIL® M and RECOMBINATE®, two therapies used to treat hemophilia A, first announced last year.

The Neil Frick Resources Center (NFRC) is excited to announce new 2026 scholarship opportunities available to the bleeding disorders community.

CSL Behring, the biopharmaceutical company behind the hemophilia B gene therapy HEMGENIX ® (etranacogene dezaparvovec-drlb), announced on March 17, 2026, a temporary global stockout of the therapy. This disruption means that some individuals in countries where HEMGENIX is commercially available may experience delays in accessing treatment.

The National Bleeding Disorders Foundation (NBDF), a leading nonprofit supporting people with bleeding disorders, is proud to congratulate Matthew Delaney, government relations manager, on being named to City & State New York’s 2026 Albany 40 Under 40, an annual list recognizing rising stars in New York State government, politics, and public service.

A Personal Connection to the Bleeding Disorders Community

Federal:  

NBDF Wraps Up Another Successful Washington Days

The National Bleeding Disorders Foundation (NBDF) is pleased to announce a new matching grant partnership with Save One Life that will double the community’s impact for children living with bleeding disorders, including hemophilia A, hemophilia B, and von Willebrand disease in developing countries.

Pathway to Cures (P2C), the venture philanthropy fund of the National Bleeding Disorders Foundation (NBDF), is pleased to announce the appointment of Michael Recht, MD, PhD, MBA, as chief medical and scientific advisor.

BioMarin Pharmaceutical Inc. has announced that it has decided to pull the hemophilia A gene therapy ROCTAVIAN (valoctocogene roxaparvovec-rvox) from the United States market.

The National Bleeding Disorders Foundation (NBDF) mourns the passing of Lynn M. Malec, MD, MSc, a physician, researchers, and champion of the bleeding disorders community.

FY 2026 Funding Update  

Last week, the House of Representatives passed the final fiscal year (FY) 2026 appropriations bills. This includes the FY26 Labor, Health and Human Services appropriations bill, which provides funding for the federal health agencies. There were big wins in the bill for the bleeding disorders community, including:  

FY 2026 Funding Update  

Last week, the House of Representatives passed the final fiscal year (FY) 2026 appropriations bills. This includes the FY26 Labor, Health and Human Services appropriations bill, which provides funding for the federal health agencies. There were big wins in the bill for the bleeding disorders community, including: