News


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Pfizer has committed to building a $100 million gene therapy manufacturing facility in North Carolina. Specifically, Pfizer will expand an 11,000-square-foot plant in Sanford, North Carolina that it acquired last year when it bought Bamboo Therapeutics, a biotech company that specializes in gene therapies for certain rare diseases. Bamboo had previously acquired the facility from the University of North Carolina.

The U.S. Food and Drug Administration (FDA) has approved Mavyret (glecaprevir/pibrentasvir), a combination drug regimen for the treatment of patients with all major genotypes (1-6) of the chronic hepatitis C virus (HCV). The once-daily, orally-administered therapy includes two distinct antiviral agents in a fixed-dose combination of glecaprevir, an NS3/4A protease inhibitor, and pibrentasvir, an NS5A inhibitor. The regimen, which is designed to last eight weeks, does not include ribavirin. Mavyret is manufactured by AbbVie.

Leading Bleeding Disorders Advocacy Organizations File Hemophilia Discrimination Complaint Against Wellmark Blue Cross and Blue Shield

Complaint argues Wellmark’s withdrawal from the Iowa marketplace discriminates against protected health conditions under the ACA, including hemophilia, and its disclosure of a patient’s personal health information violates HIPAA.

Spark Therapeutics, Inc. recently released preliminary data from a phase 1/2 dose-escalation clinical trial for SPK-8011, the company’s investigational gene therapy candidate for hemophilia A.

“Optimizing Transition to Extended Half-Life Factor Replacement Products,” is the latest educational opportunity to become available from Medscape. It is intended for hematologists, pediatricians, nurses, and other healthcare professionals who manage patients with hemophilia.

The activity is part of the online series, “Clinical Advances in Hemophilia: Management for Life,” whose purpose is to increase the knowledge, skills and competence of clinicians in providing individualized management of hemophilia.

The U.S. Food and Drug Administration (FDA) has approved expanded labeling for Epclusa®, a combination therapy for adults with chronic hepatitis C viral (HCV) infection. Manufactured by Gilead Sciences, Inc., Epclusa® encompasses two HCV therapies (Sofosbuvir/Velpatasvir) in a fixed-dose, orally administered tablet. The therapy first received FDA approval in June 2016 with an indication for all six major genotypes of the virus in patients with chronic HCV, both with and without cirrhosis. The expanded labeling will now also include patients co-infected with HIV.

The Partners in Bleeding Disorders Education Program recently launched a new online learning activity entitled, “An Overview of the Background, Inheritance and Physiology of von Willebrand Disease.”

The new module is designed to enable the learner to develop an enhanced understanding of the nature of von Willebrand disease (VWD) by providing information about the discovery and evolution of knowledge about the disease. The module also includes an explanation of the production and functions of von Willebrand factor and the varied classifications of VWD.

UniQure N.V. has announced that is has “scaled up” the manufacturing process at its large facility in Lexington, MA. According to a uniQure press release, the facility is designed to manufacture the company’s proprietary hemophilia B gene therapy candidate, AMT-060.

BioMarin Pharmaceutical, Inc. recently provided updated interim results from a phase 1/2 clinical study of BMN 270, the company’s investigational hemophilia A gene therapy. BMN 270 uses adeno-associated viruses (AAVs) as delivery vehicles, or vectors, to carry the genetic codes that prompt the production of the factor VIII (FVIII) protein that is deficient in people with hemophilia A. These AAVs are designed to deliver genetic material into living cells to sustain therapeutic effect without causing disease or triggering significant immune responses.

The Committee of Ten Thousand has requested that NHF make the public aware that their board has chosen not to participate nor support any fundraising efforts for the hemophilia memorial project, led by the National AIDS Memorial Grove.

Choose the Inscription on the Hemophilia Memorial

Vote today for the inscription that will be engraved on the Hemophilia Memorial in San Francisco, CA.

The program will allow bleeding disorders camp staff learn from one another and share best practices.

The Partners in Bleeding Disorders Education Program recently launched an updated version of “Dear Diary: The Importance of Infusion Logs in Home Infusion Therapy.” This activity aims to enable the learner to apply knowledge of the benefits and applications of paper and electronic infusion logs to support their accurate use among hemophilia patients engaged in home infusion.

Alnylam Pharmaceuticals, Inc. and Sanofi Genzyme announced positive results from an ongoing Phase 2 open label extension (OLE) study with fitusiran in patients with hemophilia A and B, both with or without inhibitors. Details from the study were presented at 26th International Society on Thrombosis and Haemostasis Meeting, July 8-13, 2017, in Berlin, Germany.

Shire Submits IND Application for Hemophilia A Gene Therapy Candidate

If effective, the therapy prompts the sustained production of FVIII and enhanced protection from bleeds.

Shire has announced the submission of an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for SHP654, an investigational factor VIII (FVIII) gene therapy for the treatment of hemophilia A.

Genentech Announces Progress in Two Phase III Emicizumab Studies

Results showed a statistically significant and clinically meaningful reduction in bleed rate, reports states.

Bleeding Disorder Organizations Unite in Opposition to BCRA

NHF, HFA, the Hemophilia Alliance and Coalition for Hemophilia B are united in their opposition to BCRA because of its damaging impact on our community and urge the Senate to reject these harmful policies.

The Senate Bill (BCRA) Will Hurt the Bleeding Disorders Community

Call TODAY and tell your Senator to vote NO on BCRA!

 

The Partners in Bleeding Disorders Education Program recently relaunched “Understanding Hemophilia,” the purpose of which is to enable the learner to understand the three major steps in normal hemostasis and the basics of treatment and management of patients with hemophilia.

The target audience includes nurses and comprehensive care staff in the U.S. hemophilia treatment center (HTC) network who are responsible for the clinical management and/or education of individuals with hemophilia.

The faculty for this module includes:

Green Park Collaborative, National Hemophilia Foundation, McMaster University, Announce Launch of CoreHEM Project

The collaboration between stakeholders to establish a core set of measurements that would be used to evaluate effectiveness of gene therapies.

The collaboration between stakeholders to establish a core set of measurements that would be used to evaluate effectiveness of gene therapies.

The collaboration between stakeholders to establish a core set of measurements that would be used to evaluate effectiveness of gene therapies.

Novo Nordisk recently announced that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for REBINYN®, the company’s recombinant, GlycoPEGylated therapy for the treatment of adults and children with hemophilia B (factor IX deficiency). A BLA is a request to the FDA for permission to introduce, or deliver for introduction, a biologic product into interstate commerce.

One of Shire’s investigational therapies, known as SHP656, failed to show significant efficacy in a phase I/II clinical study. The therapy was being developed as an extended half-life treatment for hemophilia A using proprietary technology from Xenetic Biosciences.