In a May 27th letter to the hemophilia community, it was confirmed that Spark Therapeutics has been fully integrated into the Roche Group. Spark first became a member of the Roche Group in 2019 with the aim of continuing to advance potential gene therapies for hemophilia and many other genetic diseases.

Keri Norris, PhD, JM, MPH, MCHES, Senior Vice President, Health Access and Strategic Innovation, of the National Bleeding Disorders Foundation is a Fierce DEI Award Finalist in the Leadership in Health Equity Category

Dr. Norris has been recognized for her transformative work advancing health access in the inheritable blood and bleeding disorders community.

Because May is Mental Health Awareness Month, the Neil Frick Resource Center (NFRC) is dedicating its most recent Resource Round Up (formerly HANDI Highlights) to supporting mental well-being. This edition approaches this vital topic from a variety of perspectives, with information delivered through multiple formats, including videos, articles, referral resources, and podcasts.

Through investment and partnership, Pathway to Cures encourages early-stage biotech companies to develop treatments that have the potential to improve the lives of people in our community.

Since Pathway to Cures (P2C) launched in 2023, more than 200 early-stage biotechnology, pharma, medical device and diagnostic companies, with innovative approaches that address unmet needs in our blood and bleeding disorders community, have been reviewed by our team.

Bayer recently announced that the company’s hemophilia A therapy Jivi® has been granted an expanded indication from the U.S. Food and Drug Administration (FDA). Jivi is a long-acting site-specifically PEGylated recombinant factor VIII therapy that was first FDA-approved in 2018 for use in previously treated individuals with hemophilia A, including adolescents and adults 12 years of age and over. This indication encompassed prophylactic, on-demand, and perioperative management of bleeding.

Bayer recently announced that the company’s hemophilia A therapy Jivi® has been granted an expanded indication from the U.S. Food and Drug Administration (FDA). Jivi is a long-acting site-specifically PEGylated recombinant factor VIII therapy that was first FDA-approved in 2018 for use in previously treated individuals with hemophilia A, including adolescents and adults 12 years of age and over. This indication encompassed prophylactic, on-demand, and perioperative management of bleeding.

The New England Journal of Medicine (NEJM) recently published long-term follow up data on patients who have previously received the hemophilia B gene therapy fidanacogene elaparvovec. The therapy was approved by the U.S.

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.