Novel gene editing therapies being developed for hemophilia could represent a significant advance in treatment, should they reach commercialization and become available to patients. They encompass very complex concepts and technologies that prompt new considerations relevant to long term safety and efficacy. 

The National Bleeding Disorders Foundation (NBDF) has joined with three international bleeding disorders organizations in their call to action to continue research and development into gene therapy as a treatment option for hemophilia.

Federal:

Day of Awareness on Capitol Hill for Women and Girls with Bleeding Disorders 

It has been well understood by many in the bleeding disorders community that clinical trials are, historically, very limited and not fully representative of the actual disease populations. In an effort, to drill down on the barriers to representation, the American Society of Hematology (ASH) reached out to its robust global membership to get a better sense of the challenges associated with underrepresentation in classic hematology trials. 

Welcome to the April edition of the Resource Roundup (formerly HANDI Highlights), a periodic communication of the Neil Frick Resource Center (NFRC), designed to connect the bleeding disorders community with practical and readily accessible resources. 

We need your help again! Earlier this week, the Administration announced drastic changes and reductions to the federal health programs that support the bleeding disorders community. We talked about the importance of these programs during Washington Days. We are asking you to please call or email the offices you visited in March and ask your lawmakers to reverse the cuts and protect funding for those programs.

What is the issue?

The U.S. Food and Drug Administration (FDA) recently approved Qfitlia™ (fitusiran) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors (neutralizing antibodies). The new therapy is manufactured by Sanofi.

Federal:  

Washington Days  

On March 6, over 400 advocates from the bleeding disorders community were in Washington, DC to meet with their legislators and their staff about issues important to the bleeding disorders community. Advocates participated in 248 Congressional meetings, almost evenly split between Democratic and Republican offices, representing 49 states and Puerto Rico.    

Congress Passes Bill to Fund Government Just Ahead of Shutdown Deadline 

Dear Friends,

The topic of bone health in people with bleeding disorders has received more attention and study in recent years, with reports suggesting that people with hemophilia (PwH) and people with von Willebrand disease (PwVWD) experience higher rates of osteoporosis and bone fractures. That said, screening for bone health is not consistent across federally funded hemophilia treatment centers. A lack of clear, uniform screening guidelines is a major contributing factor to this disparity.

For Immediate Release 
PRESS CONTACT:  
Kyla Clark 
National Bleeding Disorders Foundation 
347-920-0047

The Neil Frick Resources Center (NFRC) is excited to announce new 2025 scholarship opportunities available to the bleeding disorders community.

The Neil Frick Resources Center (NFRC) is excited to announce new 2025 scholarship opportunities available to the bleeding disorders community.

Welcome to the Resource Round Up (formerly HANDI Highlights), a periodic communication of the Neil Frick Resource Center (NFRC), designed to connect the bleeding disorders community with practical and readily accessible resources. 

Takeda Pharmaceuticals announced today it will globally discontinue two of its hemophilia treatments: HEMOFIL® M [Antihemophilic Factor (Human), Method M, Monoclonal Purified] and RECOMBINATE® [Antihemophilic Factor (Recombinant)]. General information is available at HemophiliaJourney.com.

Stephanie Lapidow, Executive Director of the Hemophilia Association of New Jersey (HANJ), is leading the charge to protect patients from predatory insurance practices through critical state legislation (S-3818/A-5217). In a powerful op-ed published by New Jersey Spotlight, Lapidow exposes how copay accumulator adjuster programs are leaving patients with chronic illnesses and rare disorders drowning in medical debt. These programs prevent copay assistance from counting toward out-of-pocket costs, forcing patients to pay thousands they can’t afford—while insurers double-dip.

State:
Arizona: HB 2380, a bill creating a Rare Disease Advisory Council, passed the House 46-12 on Feb. 26. 
Iowa: Bleeding Disorders of the Heartland hosted its Advocacy Day at the state capitol Feb. 5, the same day as House and Senate subcommittees were holding hearings on companion PBM reform bills HSB 99 and SSB 1074 that include accumulator adjuster bans. BDotH and NBDF testified at both hearings in support of the measures.

Hemab Therapeutics recently announced that the first patient has been dosed in their phase 1/2 clinical trial of HMB-002, an investigational subcutaneous therapy for patients with von Willebrand disease (VWD). It is developed with a monovalent antibody to increase levels of both von Willebrand factor and factor VIII. HMB-002 is a prophylactic therapy to prevent bleeding in people will all types of VWD. 

For Immediate Release 
PRESS CONTACT:  
Kyla Clark 
National Bleeding Disorders Foundation 
347-920-0047
kclark@hemophilia.org%C2%A0" title="Email for Kyla Clark">kclark@bleeding.org 

FOR IMMEDIATE RELEASE

Pfizer, Inc. has announced that they are ceasing global development and commercialization of Beqvez™, the company’s hemophilia B gene therapy product.

MEDICAL ADVISORY 
Released: February 24, 2025, 7:56 PM EST
Medical Advisory: ALTUVIIIO LOT #EY0330
 

For Immediate Release 
PRESS CONTACT:  
Kyla Clark 
National Bleeding Disorders Foundation 
347-920-0047
kclark@hemophilia.org%C2%A0" title="Email for Kyla Clark">kclark@bleeding.org 

NBDF is delighted to dedicate this edition of HANDI Highlights* to the upcoming Rare Disease Day® (RDD), which is observed every year on February 28th (or 29th in leap years) all around the world. 

? Tune In! NBDF's Bill Robie Talks HB 1216 & Rx Costs

Today, we're sharing a powerful letter from Emily Ouellette, Executive Director of the Bleeding Disorders Alliance of North Dakota (BDAND), featured on February 18, 2025, in the Minot Daily News.  

The U.S. Food and Drug Administration (FDA) recently approved Journavx™ (suzetrigine) 50 milligram 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. The drug is manufactured by Vertex Pharmaceuticals Inc, which has its North American headquarters in Boston, Massachusetts.

NBDF wants to reaffirm our unwavering commitment to our entire bleeding disorders community. Our mission—enabling every person and family affected by bleeding disorders to thrive—remains unchanged. We stand resolute in our dedication to strengthening equitable healthcare access across our diverse nation, ensuring no one faces their bleeding disorder journey alone.

The National Bleeding Disorders Foundation (NBDF) is excited to announce a new online, enduring, and accredited educational activity available to healthcare providers. The intended audience are hematologists, emergency medicine physicians, nurses, nurse practitioners, physician assistants, pharmacists, and other members of the hemophilia care team in the United States

Our hearts are with everyone affected by the devastating wildfires sweeping through Greater Los Angeles. The National Bleeding Disorders Foundation (NBDF) is actively working with local chapter leaders to support our community during this crisis. We have compiled vital emergency resources, including chapter contacts, county services, transportation assistance, and shelter information. This resource list will be updated regularly as new information becomes available.

The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the investigational von Willebrand disease (VWD) therapy VGA039. The therapy is being developed by Star Therapeutics, a biotechnology company based in San Francisco, CA.

The National Bleeding Disorders Foundation (NBDF) is pleased to announce the appointment of three new distinguished members to its Board of Directors, effective January 1, 2025. Additionally, we are delighted to announce the re-election of Susan Hartmann for another term. 

Meet Our Board

The National Bleeding Disorders Foundation (NBDF) is proud to announce its achievement of the prestigious Platinum Seal of Transparency from Candid (formerly known as Guidestar), placing NBDF among the top 0.1% of U.S.

The National Bleeding Disorders Foundation (NBDF) proudly announces that our former President and CEO, Dr. Leonard A. Valentino, has been appointed President of the World Federation of Hemophilia USA (WFH USA). This appointment represents an exciting expansion of Dr. Valentino's dedication to the bleeding disorders community, now on a global scale.

The National Bleeding Disorders Foundation (NBDF) proudly announces that our former President and CEO, Leonard A. Valentino, MD, has been appointed as President of WFH USA. This appointment represents an exciting expansion of Doctor Valentino's dedication to the bleeding disorders community, now on a global scale.

In a significant advancement for von Willebrand Disease (VWD) research and treatment, an international team of experts, including patients, clinicians, and researchers, has developed the first-ever core outcome set (COS) for VWD clinical trials. The initiative, known as coreVWD, establishes standardized outcomes that should be measured in all clinical trials for both prophylaxis and perioperative VWD treatments.

The U.S. Food and Drug Administration (FDA) has approved Alhemo (concizumab-mtci) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors.

NBDF Mourns the Loss of Dr. Marion Koerper

The National Bleeding Disorders Foundation is proud to support an important research initiative led by Emily Agen, a genetic counseling graduate student at the University of Michigan, who is investigating our community's understanding of hemophilia gene therapy treatments.

Atlanta, Georgia based Expression Therapeutics recently announced phase 1 clinical trial results based on ET3, the company’s investigational gene therapy. ET3 is developed with novel lentiviral vectors.