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In a recent press release Shire announced that they have entered into a collaboration agreement with Rani Therapeutics. This agreement allows Shire to conduct research on the use of Rani Pill™ technology for the oral delivery of factor VIII (FVIII) therapy for patients with hemophilia A. As part of the agreement, Shire obtains exclusive rights to conduct research on, evaluate, and pursue a worldwide, exclusive license for the development and commercialization of such a therapy.

Spark Therapeutics provided clinical trial updates on the company’s investigational gene therapies for hemophilia A and B, known respectively as SPK-8011 and SPK-9001. The updates were presented at the recent 59th American Society of Hematology (ASH) Annual Meeting and Exposition, which was held December 9-12, 2017 in Atlanta, GA.

NHF Healthcare Update: Congressional To-Do List

Congress has a lengthy to-do list before the end of the year.  NHF is closely monitoring numerous issues affecting the bleeding disorders community including:

Tax Reform

Genentech recently reported results from the phase III HAVEN 3 study, which showed that the prophylactic use of HEMLIBRA® led to significantly reduced bleeds in adult and adolescent patients with hemophilia A, without inhibitors. The therapy was co-developed by Genentech, Chugai and Roche.

It's Not Too Late: Take Action on Tax Reform



The National Hemophilia Foundation (NHF) is pleased to announce Christopher J.

The Partners in Bleeding Disorders Education Program recently launched “Psychosocial Considerations in Bleeding Disorder Care: Birth to Preadolescence.” This online learning activity is focused on the developmental and psychosocial issues unique to children with bleeding disorders, specifically those from infancy through preadolescence. The activity also encompasses the psychosocial considerations that may be seen in families of affected children.     

Action to Hope Study Open to Hemophilia Community

The study seeks to shed light on the barriers people with hemophilia who are obese or overweight have to losing weight.

The National Hemophilia Foundation (NHF) would like to make healthcare providers aware of three monographs that are available through NHF’s resource center HANDI. Each booklet is devoted to a key subject in bleeding disorders management, including the genetics of hemophilia, von Willebrand disease and inhibitors in hemophilia.

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. The once-weekly therapy was co-developed by Chugai, Roche and Genentech. While existing factor therapies are delivered intravenously, HEMLIBRA® is unique in that it is administered subcutaneously via an injection just under the skin.

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.