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Swedish Orphan Biovitrum (Sobi) recently announced the successful enrollment of the first hemophilia A patient in the company’s ReITIrate clinical trial. The phase 4 study, which is being sponsored by Sobi and Bioverativ Therapeutics, is designed to evaluate the use Eloctate™ for immune tolerance induction (ITI) therapy in hemophilia A patient with inhibitors. ITI is an effective approach to inhibitor eradication in which the body begins to tolerate a therapy after daily doses of factor are administered over time.

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to TRM-201 (rofecoxib), a class of COX-2 selective non-steroidal anti-inflammatory drug (NSAID) for the treatment of hemophilic arthropathy. Rofecoxib is manufactured by Cambridge, Massachusetts-based Tremeau Pharmaceuticals. A start-up company, Tremeau is focused on developing nonopioid pain treatments for rare diseases.

FDA Approves SubQ Treatment for Hemophilia A with Inhibitors

The U.S. Food and Drug Administration (FDA) has approved HEMLIBRA® (emicizumab) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with factor VIII inhibitors.

Call to Action on Senate Tax Plan

Call your Senators now and share how the tax reform proposal will harm the bleeding disorders community.

THSNA Summit Coming in March 2018

Learn about the most current clinical and scientific issues related to hemostasis and thrombosis.

In a recent press release, Alnylam Pharmaceuticals announced that the company had achieved “alignment” with the U.S. Food and Drug Administration (FDA) on safety measures and risk mitigation strategies with an eye towards resuming clinical trials of the investigational therapy fitusiran. Alnylam is developing the therapy to treat patients with hemophilia A and B, both with and without inhibitors.

The history of hemophilia treatment is marked by an evolution that has ultimately led to increased life expectancy for persons with hemophilia. By 2050, 20% of the hemophilia population will be over 60 years of age. The increase in life expectancy among persons with hemophilia has been accompanied by the emergence of conditions related to aging in the hemophilia population.

The National Hemophilia Foundation (NHF) is pleased to announce the 2017 Judith Graham Pool (JGP) Postdoctoral Research Fellowship award recipients: Esther Cooke, PhD, from the University of California San Diego, Satish Nandakumar, PhD of Boston Children’s Hospital, and Kasturi Pal, PhD, from The Scripps Research Institute in La Jolla, California.

New Educational Webinars for Healthcare Providers Outside Hematology

Primary care physicians, obstetrician/gynecologists, nurse/nurse practitioners, dentists, oral surgeons and other allied professionals are encouraged to take advantage of these excellent, accredited learning opportunities

The National Hemophilia Foundation (NHF) is pleased to announce Improving Outcomes for von Willebrand Disease and Other Bleeding Disorders in Non-Hematology Health Care Settings, a brand-new series of webinars designed to better educate healthcare providers who do not work in the field of hematology. The project is supported by independent educational grants from Shire and the Centers for Disease Control and Prevention.

 

Interim study results recently published online in the journal Haemophilia, showed that prophylactic use of ELOCTATE® led to joint health improvements in a group of hemophilia A patients. Manufactured by Bioverativ Inc., ELOCTATE®, is a recombinant factor VIII Fc fusion protein (rFVIIIFc) therapy designed to keep the infused clotting factor circulating in the body longer, stretching the time between infusions.

The U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) submitted by Bayer for BAY 94-9027, the company’s long-acting site-specific PEGylated recombinant factor VIII therapy for the treatment of hemophilia in adolescents and adults 12 years of age and over.

The U.S. Food and Drug Administration (FDA) recently granted “breakthrough” therapy designation for valoctocogene roxaparvovec (formerly BMN 270), an investigational hemophilia A gene therapy candidate developed by BioMarin Pharmaceuticals.

The Partners in Bleeding Disorders Education Program recently launched “Clinical Management of Inhibitors Module 3: Immune Tolerance Induction (ITI),” the purpose of which is to sharpen clinical knowledge and skills relating to the nursing role in eradication of inhibitory antibodies to factor VIII and IX via ITI therapy.

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Shire’s SHP654, an investigational factor VIII (FVIII) gene therapy for the treatment of hemophilia A. The therapy was formerly known as BAX 888 – Shire changed the name to SHP654 when it acquired Baxalta in June 2016.

Joint Endorsement of Alexander-Murray

Four leading bleeding disorders organizations joint together to endorse the bipartisan Alexander-Murray Market Stabilization bill.

UniQure recently announced plans to “expeditiously advance” its investigational hemophilia B gene therapy AMT-061 into a pivotal study in 2018. The announcement followed meetings the company had with both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency.

Study Finds New Information on FVIII Inhibitors

Researchers have made an important discovery that could change the way doctors understand and treat inhibitor development in hemophilia A

Actions from the President adds uncertainty to the health insurnace market.

The National Hemophilia Foundation is pleased to announce that Michael Kuklish of Lillian, Alabama, is the recipient of the 2017 Kevin Child Scholarship. 

Honoring George McCoy

A celebration of life for George McCoy will be held in Cary, North Carolina, on November 12th.

New Educational Activity for Hemophilia Providers Available

This activity discusses the latest advances in the management of hemophilia.

NHF is working with the HTC in Puerto Rico to coordinate aid to people with bleeding disorders.

San Francisco-based Catalyst Biosciences, Inc. recently announced positive clinical data from the first group of patients participating in an ongoing Phase 1/2 proof-of-concept study in individuals with severe hemophilia B. The trial, which is being conducted at three centers in South Korea, is designed to evaluate Catalyst’s investigative, subcutaneous factor IX (FIX) therapy known as CB 2679d. Catalyst develops its therapies using engineered proteases, enzymes that increase the rate of chemical reactions within cells.

NHF Research Recognized by US Specialty Pharmacists

The findings showed the value of collaboration between HTCs, insurers, and speciality pharmacies.

The mission of the Patient Reported Outcomes, Burdens, and Experiences (PROBE) study is to investigate and directly probe patient perspectives on outcomes they deem relevant to their life and care. PROBE aims to develop a new global tool to enhance the direct patient-voice in health care decision-making. The study has partners in both developed and developing countries.

Members of the National Hemophilia Foundation’s (NHF) Medical and Scientific Advisory Council (MASAC) recently gathered during NHF’s 69th Annual Meeting on August 26, 2017 in Chicago. The meeting generated three new documents related to treatment, therapies, women with bleeding disorders, carriers and emergency room management. These documents, which are listed below, were subsequently adopted by NHF’s Board of Directors on September 17, 2017.

Your Access to Healthcare is at Risk!

Call your Senator and ask them to vote NO on the Graham-Cassidy bill, which would jeopardize access to healthcare for people with bleeding disorders.

The Partners in Bleeding Disorders Education Program recently launched a new online learning activity entitled, “Bacteremia: The Good, The Bad, and The Ugly.”

Renowned Hematologist Harold R. Roberts Dies at Age 87

September 9, 2017 saw the passing of Harold R. Roberts, MD at the age of 87. Over the course of his more than 50-year career, the North Carolina native was a highly regarded physician, teacher, researcher, author and mentor in the field of hematology.

Researchers at the Boston Children’s Program in Cellular and Molecular Medicine and the Harvard Medical School recently made important discoveries relevant to the functioning of von Willebrand factor (VWF). The study, “Flow-induced Elongation of von Willebrand Factor Precedes Tension-Dependent Activation,” was published online, August 23, 2017, in the journal Nature Communications.

The U.S. Food and Drug Administration has accepted the Biologics License Application (BLA) and granted priority review for emicizumab, as a once-weekly treatment for adults, adolescents and children with hemophilia A with factor VIII inhibitors. Emicizumab, which is being co-developed by Chugai, Roche and Genentech, is unlike existing treatments in that it is administered subcutaneously via an injection just under the skin, instead of intravenously.

Emergency Preparedness

7 Ways You Can Prepare for Emergencies. Tips and resources that may help you during this hurricane season.

Alnylam Reports Patient Death in Fitusiran Clinical Study

Alnylam Pharmaceuticals, Inc. has reported on the death of a hemophilia A patient who had been participating in a Phase 2 study for the company’s investigational therapy fitusiran.

The Partners in Bleeding Disorders Education Program recently launched a new online learning activity entitled, “Genetics of Hemophilia.” The target audience for this presentation includes nurses and other health care professionals who are part of a U.S. hemophilia treatment center (HTC), who are interested in learning more about the genetic aspects of the condition.

Bayer Submits Application for Long-Acting FVIII Candidate

Bayer applied for a Biologics License Application for BAY 94-9027, the company’s long-acting site-specific PEGylated recombinant factor VIII therapy for the treatment of hemophilia A.

Sangamo Therapeutics, Inc. and Pfizer Inc. recently announced that the first patient received treatment in a phase 1/2 clinical trial designed to evaluate SB-525, the company’s gene therapy program for people with hemophilia A, or factor VIII (FVIII) deficiency. SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer for the development and commercialization of hemophilia A gene therapy programs.

The U.S. Food and Drug Administration has approved additional vial sizes for Nuwiq®, Octapharma’s recombinant FVIII therapy to treat hemophilia A patients. The new vial sizes will include 2500, 3000 and 4000 International Units (IU) and will be available in September 2017. The existing sizes include 250, 500, 1000 and 2000 IU.

Disaster Relief for Our Families

Our families in the Houston area are in need of help and here is how you can assist.

Harvey Disaster Relief for Our Families

Our families in the Houston area are in need of help and here is how you can assist.

Irma Disaster Relief for Our Families

Our families in the Florida area are in need of help and here is how you can assist.

Assistance during Hurricane Harvey

Our hearts go out to our families and staff who have been impacted by Hurricane Harvey.  Please know that we are here to help in any way we can.

Pfizer to Build Gene Therapy 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.

FDA Approves New Combination Therapy for all Major Hepatitis C Genotypes

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).

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 Reports Preliminary Data from Hemophilia A Gene Therapy Trial

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.

FDA Expands Labeling for Epclusa® to Include Co-Infected Patients with HCV/HIV and Warning for HBV Reactivation

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.

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.