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Genentech, a member of the Roche Group, today announced that the U.S. Food and Drug Administration (FDA) has approved Hemlibra® for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors (FVIII). The therapy, which was co-developed by Genentech, Chugai and Roche, was already FDA approved in the fall of 2017 for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with FVIII inhibitors.

The National Hemophilia Foundation (NHF) is pleased to announce the 2018 NHF-Shire Clinical Fellowship award recipients: Hanny Al-Samkari, MD, of Boston Hemophilia Center/Massachusetts General Hospital and Harvard University, and Michael H. White, MD, of Emory University and Children’s Healthcare of Atlanta (CHOA).

Fall is upon us as crisper, cooler weather prevails across much of the country. It is arguably the season most evocative of change, and in that spirit NHF’s information resource center HANDI would like to share several resources that may offer guidance as you or a family member face a major life transition.

HANDI hopes that you find these resources to be helpful and informative!

Copay Accumulator Patient Impact

Watch this video to learn how Copay accumulator adjustments affect patients requiring specialty medications with no generic alternatives.

The National Heart, Lung, and Blood Institute (NHLBI) is currently seeking public feedback that will inform future research the agency will conduct on inhibitors to factor VIII (FVIII) in patients with hemophilia A. To this end, the NHLBI has released a Request for Information (RFI) to solicit perspectives and comments from the bleeding disorders community on potential strategies, scientific opportunities and priorities, and the requisite infrastructure to optimize inhibitor research.

As individuals with hemophilia live longer and reach life expectancy rates comparable to the general population, their healthcare providers will continue to encounter clinical challenges inherent in treating and managing aging patients, including cardiovascular disease (CVD). There are several established risk factors associated with CVD such as hypertension, overweight, obesity and an abnormal lipid profile.

Medscape has launched a new educational activity in their “Clinical Advances in Gene Therapy for Hemophilia” series, which is presented through a collaboration between Medscape, the National Hemophilia Foundation, the European Haemophilia Consortium and the World Federation of Hemophilia. The program is supported by an independent educational grant from BioMarin.

Bayer receives FDA approval for hemophilia A treatment

Bayer announced today that the US Food and Drug Administration (FDA) has approved Jivi® (BAY94-9027), the company’s long-acting site-specifically PEGylated recombinant factor VIII therapy for the treatment of hemophilia A in adolescents and adults 12 years of age and over. This treatment has been approved for prophylactic, on-demand and perioperative management of bleeding in this population.

Bayer receives FDA approval for hemophilia A treatment

Bayer announced today that the US Food and Drug Administration (FDA) has approved Jivi® (BAY94-9027), the company’s long-acting site-specifically PEGylated recombinant factor VIII therapy for the treatment of hemophilia A in adolescents and adults 12 years of age and over. This treatment has been approved for prophylactic, on-demand and perioperative management of bleeding in this population.

The new vial size will reduce the reconstitution time that was needed to prepare multiple vials for a similar dose.

The therapy is for people with severe and moderately severe hemophilia B.

Catalyst Biosciences recently announced updated positive interim data from a phase 2/3, multinational clinical study of marzeptacog alfa (MarzAA), a subcutaneously administered factor VIIa therapy. MarzAA is being developed by Catalyst for the prophylactic treatment of individuals with hemophilia A or B with inhibitors.

NHF 70th: Pursuing discovery of the next generation of therapies

An interview with one of the recipients of NHF's Innovative Investigator Awards

NHF 70th: Pursuing discovery of the next generation of therapies

An interview with one of the recipients of NHF's Innovative Investigator Awards

Sangamo Therapeutics, Inc. recently announced positive preliminary data from the 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.

Spark Reports Progress on Hemophilia A Gene Therapy Candidate

The therapy is administered via a one-time intravenous infusion, which is designed to elicit the production of therapeutic levels of factor VIII.

Spark Therapeutics recently reported preliminary phase 1/2 data for SPK-8011, the company’s investigational gene therapy candidate for hemophilia A. The therapy is administered via a one-time intravenous infusion, which is designed to elicit the production of therapeutic levels of factor VIII (FVIII), a protein that is normally deficient in individuals with hemophilia A. Spark Therapeutics’ proprietary bioengineered adeno-associated viruses (AAVs) act as delivery vehicles, or vectors, to carry the genetic codes that prompt the FVIII production.

Court Rules on Shire/Roche Preliminary Injunction

The preliminary injunction was part of an ongoing patent lawsuit.

Medscape has launched a new educational activity in their “Clinical Advances in Gene Therapy for Hemophilia” series, which is presented through a collaboration between Medscape, the National Hemophilia Foundation, the European Haemophilia Consortium and the World Federation of Hemophilia. The program is supported by an independent educational grant from BioMarin.

“The Science of Gene Therapy for Hemophilia,” will examine the technology behind the emerging approaches to gene therapy in hemophilia.

Upon completion of this activity, participants will be able to:

Shire is discontinuing the manufacture and distribution of BEBULIN®, the company’s factor IX human plasma-derived prothrombin complex concentrate product. BEBULIN® is indicated for the prevention and control of bleeding episodes in adult patients with hemophilia B. The National Hemophilia Foundation has been notified by Shire that this decision was based on the reduced demand and is not connected with any new safety or efficacy findings in relation to the therapy.

Healthcare providers interested in broadening their knowledge of the science and clinical management of inhibitors are enthusiastically invited to participate in NHF’s webinar on “Current Research and Future Approaches to Eradicating Inhibitors in Hemophilia.” This CME and CNE-accredited activity reviews the basic science behind the development of inhibitor antibodies to treatment for hemophilia, the associated management complications and current barriers to research.

A new patient financial assistance program has become available for individuals with hemophilia. The Assistance Fund (AF) is a charitable foundation that was created to provide eligible patients with chronic diseases help with paying medication copayments, coinsurance, deductibles, insurance premiums and incidental medical expenses.

As “the dog days” of August approach, Autumn may still seem a long way off. Though with a quick glance of the calendar, we see that colleges begin their class terms earlier and earlier and Fall semester is right around the corner! Whether going away or staying closer to home, being a first-year college student can be a time fraught with great excitement, anticipation and perhaps some trepidation about this next big step. HANDI, NHF’s information resource center, would like to highlight several resources aimed at helping new college students in this transition.

NHF Comments on Trump Administration Drug Pricing Blueprint

NHF submitted a letter on the "American Patients First" drug pricing blueprint.

HTRS

The HTRS MRA provides financial support for qualified fellows or junior attending/junior faculty pursuing clinical, translational, or basic science research in hemostasis and/or thrombosis under the guidance of an experienced mentor.