Early Treatment Linked to Reduced Joint Damage in JOS-C Study
After 26 years of research, including by many members of our HTC staff, results show that starting prophylaxis treatment earlier leads to better outcomes for patients with severe hemophilia A. The Joint Outcome Continuation Study built on previous research and a follow up study of children up to age 18. See details at ashpublications.org.
NHF Switches Annual Conference to Online Virtual Resource
The National Hemophilia Foundation has changed their Annual Bleeding Disorders Conference to be a fully online, virtual interactive experience. The decision to switch to a virtual experience was in response to the COVID-19 pandemic in an effort to keep the bleeding disorders community safe. See full article at hemophilia.org.
BioMarin's Gene Therapy for Hemophilia A Remains on Track for US Approval
BioMarin's Application to the FDA for approval of their experimental gene therapy for adults, valoctocogene roxaparvovec, remains on track despite the outbreak of the COVID-19 pandemic. The company announced that if approved, the gene therapy will be labeled Roctavian instead of Valrox. See full article at hemophilianewstoday.com.
Study Finds Prophylaxis is Better than On-Demand for Adults with Hemophilia A
A recent study of German and Hungarian adult patients with Hemophilia A, and published in Haemophilia, indicates that patients who used prophylactic treatment fared better than those who only sought treatment for bleeds on an on-demand basis. See full article at hemophilianewstoday.com.
NHF Colorado Will Host Family Campfire in Virtual Online Activity
In reaction to the restrictions to gatherings due to the COVID-19 pandemic, NHF Colorado has shifted many of their annual activities to virtual events. The first such event will replace the usual Family Camp with a virtual Campfire event on Jun 5, 2020. Changes to Mile High Camp are also now in place. See more information at cohemo.org.
New Bloodstream Media Podcast Tackles Pain
Bloodstream Media has created a new podcast that specifically looks at pain. Specialists in hemophilia pain management, including our HTC's Dr. Tyler Buckner, participate in answering questions and overcoming the challenges of dealing with chronic pain and pain from bleeds. Listen or link to the first episode at bloodstreammedia.com.
Backpacks + Bleeders Offer "Trailblaze from Home" Challenge
In order to encourage patients and families to keep exercising, stay fit, and also stay at home, Backpacks + Bleeders has a fun new program designed to reward participants for their stay at home efforts. Rewards include patches, stickers, shirts and more. Each month there are new updates and ways to earn points. See details here.
NHF Colorado and HTC Share Updates on COVID-19 in Virtual Town Hall
NHF Colorado and HTC staff members shared updates and information on the COVID-19 pandemic, how we are responding, and what support is available to patients and families in a virtual town hall held on April 9, 2020. The town hall was available to view live and is now available to watch online. Registration is required to view here.
FDA Approves Sevenfact for Hemophilia A and B With Inhibitors
The FDA has approved Sevenfact for the treatment and control of bleeding episodes occurring in adults and children ages 12 and up with hemophilia A or B who have inhibitors. The new treatment uses an active ingredient expressed in genetically engineered rabbits. See full article at fda.gov.
Medexus Acquires IXINITY from Aptevo
Aptevo Therapeutics has sold their recombinant factor IX product, IXINITY to Canada based Medexus Pharmaceuticals. IXINITY is used for the control and prevention of bleeding episodes and for perioperative management in adults and children 12 and older with hemophilia B. See full article at hemophilia.org.
Survey of Healthcare Providers Shows Obesity a Likely Factor in Joint Bleeds
A survey of US Healthcare providers showed that obesity is likely a factor in joint bleeds and pain for hemophilia patients. Dr. Mike Wang from our HTC was one of the lead researchers on this project that worked with providers to rate the levels o concern about weight and its impact on health of their patients. See full article at hemophilianewstoday.com.
MASAC Addresses Concerns about Coronavirus and Bleeding Disorders
NHF's Medical and Scientific Advisory Council (MASAC) issued a letter to the bleeding disorders community addressing concerns about the COVID-19 virus (Coronavirus) that is spreading worldwide. The letter addresses keeping dosing of factor on hand, supply chain issues, and safety of plasma products. See full article at hemophilia.org.
Valrox, Gene Therapy for Hemophilia A, Accepted for Priority Review by FDA
The FDA has accepted, under a priority review, the marketing application for Valrox, an investigational gene therapy for hemophilia A from BioMarin. The gene therapy is the first accepted by the FDA that is specifically intended to treat hemophilia. Because it was granted priority review, a decision regarding the approval is expected by late August of 2020. See full article at hemophilianewstoday.com.
Novo Nordisk Launches Esperoct, Long-Acting Recombinant for Hemophilia A
Novo Nordisk announced the launch of Esperoct, the company's long-acting recombinant factor VIII product for the prevention and treatment of bleeding in patients with hemophilia A. The new therapy can be used for routine prophylaxis to reduce frequency of bleeding, on-demand treatment, control of bleeding episodes and perioperative management of bleeding. See full article at hemophilia.org.
Bloodstream Media Interviews Dr. Laura Fox, PT in Bloodstream MINI
Bloodstream Media, who provide multiple podcasts about bleeding disorders, recently chose Dr. Laura Fox, our lead Physical Therapist, to introduce their MINI series- smaller interviews for their Bloodstream podcast. Laura talks about physical therapy and bleeding disorders with host Patrick James Lynch. Listen at podcasts.apple.com.
Study Suggests VWD Primary Postpartum Hemorrhage Remains a Challenge
A recent study at Thomas Jefferson University in Philadelphia, PA suggests that primary postpartum hemorrhage (PPH) is a persistent clinical challenge in patients with type 3 von Willebrand disease (VWD). The study showed that even those who received a diagnosis before pregnancy still had problems after birth. See full article at hemophilia.org.
FDA Updates Guidelines for Gene Therapy Treatments
The FDA announced six final guidelines on gene therapy manufacturing and clinical development of products, as well as a draft guidance regarding gene therapies and orphan drugs. The FDA anticipates many more gene product approvals in coming years. The guidelines should give developers recommendations on manufacturing issues, safety and effectiveness standards. See full article at centerforbiosimilars.com.
Dr. Leonard A. Valentino Chosen as the New President and CEO of NHF
The board of directors at the National Hemophilia Foundation (NHF) announced Dr. Leonard A. Valentino will be the new Chief Executive Officer. Dr. Valentino will begin as CEO on February 17, 2020 and replaces Val Bias, who retired in late 2019 after more than a decade in that role. See full article at hemophilia.org.
BioMarin Says Gene Therapy May Cost up to $3 Million
BioMarin, who is currently progressing to approval of Valrox (valoctocogene roxaparvovec), gene therapy treatment for hemophilia A, is considering pricing the treatment at $2 million to $3 million. The price range would set Valrox as the world's most expensive one-time therapy, but it could save healthcare systems more than $20 million over a typical patient's lifetime. See full article at hemophilianewstoday.com.
Sangamo Transfers Development of SB-525 Gene Therapy to Pfizer
Sangamo Therapeutics has handed over development of hemophilia A gene therapy candidate SB-525 to Pfizer. The therapy had been developed by Sangamo in collaboration with Pfizer and completed two of three clinical trails. Pfizer will be advancing to phase 3 clinical trials. See full article at hemophilia.org.
Emicizumab May Yield Improvements in Joint Health in Hemophilia Patients
Results presented at the American Society of Hematology (ASH) showed that emicizumab may yield clinically relevant improvements in joint health in patients with hemophilia. The results indicate that emicizumab has a favorable safety profile and has led to reductions in joint bleeds. See full article at hematologyadvisor.com.
UniQure and Pfizer Share Updates on Gene Therapy Trials for Hemophilia B
Both UniQure and Pfizer shared updates on their gene therapy trials for hemophilia B patients at the annual American Society of Hematology (ASH) national meeting in Orlando, Florida. Both trials have been ongoing for more than a year and both show promise. See full article at biopharmadive.com.
Takeda and Enzyre Partner to Develop Home Blood Test for Hemophilia Patients
Enzyre is teaming up with Takeda to develop a diagnostic device that will allow people with hemophilia to determine their blood coagulation status at home. The goal is to create a device that enables patients to determine their status in nearly real time and share results with treating physicians through a mobile app. See full article at hemophilianewstoday.com.
Study Examines Adherence Rates for Hemophilia Patients Using Emicizumab
A new study shared at the American Society of Hematology (ASH) national meeting in December, found that patient with hemophilia who require routine prophylaxis may be more adherent to emicizumab than to previous factor or bypass agents. The study aimed to asses adherence rates and find potential predictors of nonadherence associated with emicizumab treatment. See full article at pharmacytimes.com.
Sangamo Shares Data for Gene Therapy Study on SB-525 for Hemophilia A
Sangamo Therapeutics and Pfizer announced updated follow-up results from their Phase 1/2 Alta study evaluating investigational SB-525 gene therapy in patients with severe hemophilia A. The data showed that it was generally well tolerated, and demonstrated sustained increased Factor VIII levels following treatment through to 44 weeks. See full article at businesswire.com.
Updated MASAC Guidelines on Emergency Department Management Approved
The National Hemophilia Foundation's Medical and Scientific Advisory Council (MASAC) has issued an important new document on emergency department management, which was adopted by the NHF's Board of Directors on Dec 5, 2019. The document includes guidelines including triage, assessment, and diagnostics for patients who need to visit the emergency department. See full article at hemophilia.org.