News

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Be sure to check back regularly to get our latest news updates.


NHF's Wednesday Webinars for April 2021 will feature an array of speakers and topics of interest to both healthcare providers and patients and families with blood disorders alike.

April Webinars
All webinars are from 2:00pm-3:00pm ET.

April 7th, 2021
What is the Burden of Problem Joints with Hemophilia?

Speaker: Paul McLaughlin

April 14th, 2021
WFH Humanitarian Aid Program

Speaker: Assad Haffar

The American Rescue Plan (ARP), signed into law on March 11, 2021, provides new opportunities to get health insurance coverage, and/or to reduce the cost of your premiums. Review your coverage now to make sure you are maximizing your benefits under the new law.

 

uniQure recently announced the results of a comprehensive investigation into a case of hepatocellular carcinoma (HCC) diagnosed in one patient participating in the phase III HOPE-B pivotal trial of etranacogene dezaparvovec, the company’s investigational gene therapy candidate for individuals with severe and moderately severe hemophilia B.

uniQure recently announced the results of a comprehensive investigation into a case of hepatocellular carcinoma (HCC) diagnosed in one patient participating in the phase III HOPE-B pivotal trial of etranacogene dezaparvovec, the company’s investigational gene therapy candidate for individuals with severe and moderately severe hemophilia B.

To the members of the inheritable blood disorders community,

Let me begin with a word for the thousands of members of our community – patients, providers, partners, family, and friends – who are also members of the Asian and Pacific Islander community:

We would not be the National Hemophilia Foundation without you.

As part of a patient education webinar series known as ITP INSIGHTSSM, the Platelet Disorder Support Association (PDSA) recently recorded a session on the new COVID-19 vaccines and immune thrombocytopenic purpura (ITP), a disorder characterized by low platelet levels.

The National Hemophilia Foundation (NHF) announced that Keri L. Norris, PhD, MPH, MCHES, has joined NHF as  vice president of health equity, diversity, and inclusion. Dr. Norris has more than 20 years of experience in health equity, public health, and social justice, with a particular emphasis on health disparities. Her role will be to create a health equity framework to integrate into NHF programs and services and develop culturally and linguistically appropriate programs and services to address disparities in outcomes within our community.

The National Hemophilia Foundation (NHF) announce plans for a NHF State of the Science Research Summit to bring together the inherited bleeding disorders community for a virtual conference to identify and accelerate research progress in the areas of greatest need for affected individuals and their families. The event is scheduled to be held virtually September 12-15, 2021.

In Genentech’s ongoing efforts to transparently communicate with the hemophilia community, we are sharing two updates to the Hemlibra label that were requested by the FDA. These changes, which were accepted and implemented on March 10, 2021, impact the Warnings and Precautions (sections 5.1 and 5.2) and Clinical Pharmacology (section 12.3) sections of the label.​ Importantly, the overall benefit/risk profile of Hemlibra remains positive.

The Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation (NHF) issued three new documents, all of which were adopted by NHF’s Board of Directors on March 4, 2021.

The Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation (NHF) issued three new documents, all of which were adopted by NHF’s Board of Directors on March 4, 2021.

The National Hemophilia Foundation (NHF) is refocusing its mission statement to reflect a renewed emphasis on research and an expanded scope to include inheritable blood disorders.

The newly adopted mission statement reads:

The mission of the National Hemophilia Foundation is dedicated to finding cures for inheritable blood disorders and to addressing and preventing the complications of these disorders through research, education, and advocacy, enabling people and families to thrive.

The National Hemophilia Foundation is now accepting nominations for its Awards of Excellence. These awards honor members of the bleeding disorders community who have made outstanding contributions to improving the lives of people with bleeding disorders through treatment and care, advocacy, and raising awareness.

The Children’s National Hospital and Takeda recently announced the creation of a new, first-of-its-kind program to help standardize and streamline the process of diagnosis and care for individuals with rare diseases.

NHF's Wednesday Webinars for March 2021 will feature an array of speakers and topics of interest to both healthcare providers and patients and families with blood disorders alike.

March Webinars
All webinars are from 2:00pm-3:00pm ET.

 

March 3, 2021

Cellular Therapies for Hemophilia 

Speakers: Amy Shapiro, MD, Indiana Hemophilia & Thrombosis Center, Inc. and Deya Corzo, MD, Sigilon Therapeutics

 

The U.S. Food and Drug Administration (FDA) has partially rescinded a pair of supplemental Biologics License Applications (BLAs) the agency had granted, in error, for two recombinant factor IX products, BeneFIX® (Pfizer) and IXINITY (Aptevo). The supplemental BLAs in question were both approved by the FDA in 2020 and represented additional indications that ultimately ran against exclusivity rights of another product.   

Background:

Fitusiran is an RNA drug that can prevent antithrombin protein from being made in the liver. Antithrombin is an important brake for the coagulation system. Insufficient amounts of circulating antithrombin in normal adults lead to thrombosis. In persons with hemophilia (PWH) A or B, who don’t have sufficient clotting, lowering antithrombin with fitusiran has been shown to increase coagulation and protect PWH from bleeding. It is presently in Phase 3 clinical trials.

Octapharma recently announced the final results from the NuProtect study, which evaluated the immunogenicity of Nuwiq® in previously untreated patients (PUPs) with severe hemophilia A.Nuwiq® is a recombinant factor VIII therapy, is indicated for on-demand treatment and control of bleeding episodes and routine prophylaxis to reduce the frequency of bleeding episodes and perioperative m

Spark Therapeutics announced preliminary data from part one of their ongoing phase 1/2 open-label, non-randomized, dose-finding study of SPK-8016, the company’s investigational gene therapy for hemophilia A.

Come the Spring of 2021 patients and caregivers will be presented with a new opportunity to provide valuable feedback on the care they receive at hemophilia treatment centers (HTC) across the United States.

NHF's Wednesday Webinars for February 2021 will feature an array of speakers and topics of interest to both healthcare providers and patients and families with blood disorders alike.

February Webinars
All webinars are from 2:00pm-3:00pm ET.

On January 28th, 2021, President Biden signed an executive order aimed at stabilizing and strengthening the Affordable Care Act (ACA) and Medicaid. The President specifically stated that this order does not change any law but rather rolls back actions taken by the Trump administration over the last four years.

BioMarin recently announced positive updates from its ongoing phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. The trial currently includes 134 participants, all of whom have received a single dose of the therapy and have been observed for at least one year post treatment.

CSL Behring has announced a voluntary pharmacy level product recall of one batch of Mononine Coagulation Factor IX (Human), a plasma-derived product used to treat hemophilia B. CSL Behring’s notification states, “Patients can continue to use product they may have. Although the potential for safety risk to patients is considered low, it cannot be fully excluded.”

The affected batch is identified as: P100125501

The new joint clinical practice guidelines on the diagnosis and management of von Willebrand disease, developed by the American Society of Hematology (ASH), the International Society of Thrombosis and Haemostasis (ISTH), the National Hemophilia Foundation (NHF), and the World Federation of Hemophilia (WFH) now have snapshot guides available in English, Spanish, and French.

The snapshots provide a high-level summary of both the diagnosis and management of VWD.

 

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