BioMarin Announces First Patient Dosed in Gene Therapy Study for Hemophilia A Patients with AAV5 Antibodies
BioMarin Pharmaceutical announced that it has dosed the first patient in a Phase 1/2 Study evaluating its investigational gene therapy valoctocogene roxaparvovec in severe hemophilia A patients with pre-existing AAV5 antibodies. The trial is the first that is testing to see how gene therapy can benefit those who have antibodies to the vector. See full article at prnewswire.com.
Takeda Secures Takeover of Shire with $62 B Deal
Takada Pharmaceutical will pay $62 billion to acquire the rare disease biotech company Shire, in a deal that will create one of the largest drug makers by sales. The takeover was reached after Shire rejected four previous offers. A quarter of Shire's revenues in 2017 came from hemophilia medication. See full article at biopharmadive.com.
Study Suggests Prophylaxis Could Reduce Hospitalizations for VWD Patients
Researchers in Sweden and the United States published an article focused on potential correlations between patients with von Willebrand disease (VWD), hospitalizations and prophylaxis. The retrospective study investigated the frequency of hospital admittance to determine whether a prophylactic treatment regimen was associated with a reduction of hospitalizations. See full article hemophilia.org.
Sharon Funk Discusses PT and Bleeding Disorders in Ask the Expert Podcast
Our expert Physical Therapist, Sharon Funk, was interviewed in the Ask the Expert Podcast in April. She answered questions and shared her experience and insights on joint disease that she has gained throughout the years treating people with bleeding disorders. The 44 minute episode is available at bloodstreamexpert.com.
FDA Grants Breakthrough Therapy Designation for Hemlibra
The FDA has granted Breakthrough Therapy Designation for Hemlibra (emicizumab-kxwh) for hemophilia A without factor VIII inhibitors. This designation was based on results from a Phase III trial. The treatment is being watched very carefully, both for its success and for safety issues. Five deaths were reported during the trials but were attributed to unrelated causes. See full article at biospace.com.
Dr. Marilyn Manco-Johnson Talks Gene Therapy in Ask the Expert Podcast
Our HTC Director, Dr. Marilyn Manco-Johnson, sat down with Chris Bombardier, Host of the podcast "Ask the Expert" and shared her insights and knowledge of gene therapy. The concept has been there for decades, but recent clinical trials is leading to a more positive view of what it may mean in the future for those with bleeding disorders. Listen to this informative podcast at bloodstreamexpert.com.
CSL Behring Discontinues Monoclate-P
CSL Behring has announced that due to patients transitioning from plasma-derived to recombinant products, it has been difficult to sustain production and distribution of their Monoclate-P. They have discontinued the product and estimate existing supplies will only be available until December 2018. If you are currently using this product, please contact your provider. See full article at hemophilia.org.
Sharon Funk and Lynn Magnuson honored for Years of Service with CU Medicine
One of our HTC Physical Therapists, Sharon Funk and our accountant, Lynn Magnuson, were honored at a breakfast for longtime employees with CU Medicine. Lynn has worked through CU Medicine for 15 years and Sharon marked 40 years, all with patients at the HTC. See full article at cuanschutztoday.org and an article in our March 2018 newsletter here.
Miracle of Hemophilia Drugs Comes at a Steep Price
The medications required to allow those with hemophilia and other bleeding disorders to live relatively normal lives are expensive and the formulas to bring those costs down don't seem to be working. NPR examines the high costs of factor medication and the issues surrounding the pharmaceutical companies, insurance companies and patients. See full article at npr.org.
Blood Journal Article Indicates Hemophilia Gene Therapy is Effective and Safe
An Article published in the journal Blood reviews adeno-associated virus-5 (AAV5) liver-directed gene therapy in severe and moderate hemophilia B and found that it is clinically effective. The procedure examined was well tolerated and no immune responses were detected. See full article at bloodjournal.org.
NHF Helps Launch Campaign to Encourage Patients to Take Survey on HTC Care
The Second National Patient Satisfaction Survey of HTCs is underway. HTCs from around the country are sending out surveys to patients asking them how satisfied they were with their care in 2017. The survey is available on paper, in Spanish and English and can be taken online. The information gathered will help HTCs monitor their efforts. See full article at hemophilia.org. Take the survey at htcsurvey.com.
Barry Haarde, Hemophilia Advocate and Inspiration, Passes Away at 52
Much loved advocate for those with hemophilia, Barry Haarde has passed away at age 52. Barry was the founder of "Wheels for the World" campaign where he rode over 20,000 miles across the United States generating over $250,000 for Save One Life. His advocacy extended to inspiring many despite many medical issues he faced. See full article at blog.kelleycom.com.
Babies Who Suffer Stroke Regain Language Function in Opposite Side of Brain
A new study led by Georgetown University Medical Center has found that babies who experience stroke have the ability to use the right side of the brain for language. The impact of a stroke is less in infants due to how plastic brain function is. This data has been found in previous research but this study confirms it in a carefully controlled study. See full article at sciencedaily.com.
Hemophilia Gene Therapies Show Promise
Early results from trails of investigational hemophilia A and B gene therapies were published in the New England Journal of Medicine. Both trials showed substantial increases in clotting factor activity and reductions in bleeding. The two trials are being implemented by Spark Therapeutics and Pfizer. BioMarin is also involved in gene therapy trials that show promise. See full article at jamanetwork.com.
Catalyst Biosciences Given Permission to Change Trial Length for CB 2679d
Catalyst Biosciences has amended its protocol of a Phase 1/2 clinical trial investigating the factor IX variant CB2679d in previously treated hemophilia B patients. The amendment moved one of the trial groups directly into dosing. The trial is being conducted in three sites in South Korea. See full article at hemophilianewstoday.com.
NY Times Shares Patient Voices: Hemophilia
As part of a series of audio and visual presentations designed to bring awareness and personify medical disorders, the New York Times has a section they have dedicated to several members of the bleeding disorder community. Each has pictures and audio recorded by the individual talking about the impact bleeding disorders have played in their lives. See full article at nytimes.com.
Alnylam and Sanofi Partner to Treat Rare Disorders
Alnylam, an RNAi therapeutics company, and Sanofi announced a strategic restructuring of their alliance to streamline development and commercialization of some products for rare disorders. Specifically Sanofi will obtain rights to develop fitusiran to treat hemophilia A and B. See full article at hemophiliafed.org.
Chris Bombardier Completes the Seven Summits by Climbing Mt. Vinson
Chris Bombardier, a member of our local hemophilia community, has completed the Seven Summits by climbing Mt. Vinson in Antarctica on January 6th. Chris has now climbed the highest peak on all seven continents, something that only 400 people have ever done, becoming the first person with severe hemophilia to accomplish the feat. See his post at his Facebook page here.