New Section of Memorial Dedicated to People with Hemophilia who Died of AIDS
A new section of the National AIDS Memorial Grove has been dedicated to the lives of those with hemophilia who died of AIDS. The memorial is located in Golden Gate Park in San Francisco and hosted a ceremony to honor the lives of the estimated 10,000 hemophiliacs who died as a result of the virus in tainted blood supply products. See full article at sfchronicle.com.
"How do you thank someone for saving your life?"
Do you know the symptoms of thrombotic events? The Boston Globe wrote an article telling how a doctor who was attending a seminar recognized symptoms the speaker was experiencing as a potential pulmonary embolism. The doctor's knowledge and experience helped save a life. See full article at bostonglobe.com.
Alynlam's Hemophilia Program on Hold Following Patient Death
Alynlam's hemophilia clinical program that is testing fitusiran in patients with hemophilia A or B has been put on hold following the death of a patient in one of the company's trials. The company is suspending all clinical trials with fitusiran while they examine the evidence and determine whether or not the drug is associated with causing the death. See full article at raredr.com.
Catalyst Biosciences Announces Positive Clinical Data for Hemophilia B Trial
Catalyst Biosciences announced positive clinical data from the first Cohort of its ongoing Phase 1/2 proof-of-concept clinical trial in people with severe hemophilia B. The results support that CB 2679d has the potency advantage needed to be delivered as a convenient subcutaneous prophylactic therapy to prevent spontaneous bleeds. See full article at globenewswire.com.
Bioverativ and Bicycle Join to Develop Drugs for Hemophilia
Bicycle Therapeutics has partnered with Bioverativ to develop a new class of drugs for patients with rare blood disorders including hemophilia. The partnership will look to discover and select candidates for programs that will use the Bicycle Therapeutics technology called bicycles-inspired by their structure to optimize target specific therapies. See full article at labiotech.eu.
Bayer Submits BLA for Long-Acting Factor VIII for Hemophilia A
Bayer announced that they have submitted to the FDA a Biologics License Application (BLA) for BAY94-9027, a long-acting site-specifically PEGylated recombinant human Factor VIII designed to treat hemophilia A up to once every seven days. The BLA is supported by positive data from their Phase III PROTECT VIII trial. See full article at genengnews.com.
Pfizer Launches Innovative Technologies for Hemophilia Patients
Pfizer announced the launch of two first-of-its-kind innovative technologies for patients with hemophilia at the 69th Annual National Hemophilia Foundation Meeting in Chicago this August. Hemocraft, a modified version of the video game Minecraft, and HemMobile Striiv Wearable, a custom wristband designed to assist people with hemophilia, were on display during the meeting. See full article at raredr.com.
FDA Grants Priority Review of Genentech's Emicizumab for Hemophilia A
Genentech announced that the FDA has accepted their Biologics License Application (BLA) and granted Priority Review for emicizumab prophylaxis as a once-weekly subcutaneous treatment for adults, adolescents and children with hemophilia A with factor VIII inhibitors. See full article at businesswire.com.
NHF, HFA and Hemophilia of Iowa File Hemophilia Discrimination Complaint
Leading bleeding disorder organizations the National Hemophilia Foundation and the Hemophilia Federation of America joined Hemophilia of Iowa to file a legal complaint against Wellmark Blue Cross, Blue Shield. The action accuses Wellmark of violating the ACA and HIPAA laws and discriminating against patients in Iowa with hemophilia. See full article at prnewswire.com.
Hemophilia B Pediatric, Adult Patients Benefit from Idelvion, Studies Show
Results of studies CSL Behring's Idelvion shared at the 26th Biennial ISTH Congress in Berlin revealed positive findings for both adults and children with hemophilia B. The four studies cited indicated that Idelvion provided lower bleeding rates and higher quality of life for patients. See the full article at hemophilianewstoday.com.
Regis Jesuit's Elijah Warren Eager to Get Going at Arizona State
Elijah Warren, a local swimmer, and patient at our HTC, has signed to attend Arizona State University next fall. A senior this year at Regis Jesuit, Elijah has committed to swim for ASU and train under Bob Bowman, who trained Olympian Michael Phelps. Elijah has severe hemophilia and has worked hard to become a competitive swimmer on a national level. See the full article at aurorasentinel.com.
UniQure Presents Data on Hemophilia B Gene Therapy Research
Clinical data from uniQure was presented at the Congress of the International Society on Thrombosis and Hemostasis (ISTH) held in Berlin, Germany in early July. The presentation demonstrated that the presence of pre-existing anti-AAV5 neutralizing antibodies does not predict the potential efficacy of AAV5 mediated gene transfer in patients with hemophilia B. See the full article at pharmiweb.com.
BioMarin, Spark, Raising the Stakes for Hemophilia
While gene therapy is a promising new research area, BioMarin and Spark both presented data from trials at the recent International Society on Thrombosis and Hemostasis (ISTH) 2017 Congress in Berlin during July. Additional data on other upcoming treatments for hemophilia were also presented. See the full article at biopharmadive.com.
BioMarin Announces Results from Study of BMN 270 to Treat Hemophilia A
BioMarin announced an update to its previously reported interim results of a Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. The results show an annual bleed rate reduction of 97%. BioMarin plans to initiate a Phase 3 trial later this year. See the full article at streetinsider.com.
Promising New Data from Alnylam and Sanofi for Fitusiran
An experimental hemophilia drug from Alnylam that was developed in a partnership with Sanofi has promising new results. Data from a year long clinical trial show that 48% have not had any bleeding events. Before the study patients were reporting a median of 20 bleeding events per year. See the full article at statnews.com.
New England Journal of Medicine Publishes Latest Update on the HAVEN 1 Trial
The New England Journal of Medicine published an article reviewing results of a study of prophylactic treatment of hemophilia A patients with inhibitors with Emicizumab, also known as ACE910. The results showed those treated had a bleeding rate that was 87% lower than those with no treatment. See the full article at nejm.org.
FDA Approves Bevyxxa to Prevent VTE in At-Risk Patients
The FDA has approved Bevyxxa (betrixaban) as the first and only anticoagulant for hospital and extended duration prophylaxis of venous thromboembolism (VTE) in adult patients hospitalized for an acute medical illness and who are at risk for thromboembolic complications. See the full article at globenewswire.com.
Shire Submits Application to the FDA for Gene Therapy to Treat Hemophilia A
Biotech firm Shire announced that they have submitted an application to the FDA for a gene therapy drug that treats hemophilia A. SHP654, which was also designated as BAX 888, was submitted based on pre-clinical and Phase 1 studies that showed promise for both hemophilia A and B patients. See the full article at iii.co.uk.
FDA Accepts Investigational New Drug Application for Hemophilia A Treatment
The FDA accepted Bioverativ's Investigational New Drug (IND) application for their hemophilia A therapy BIV001. The treatment is currently the only molecule designed to fuse 4 different proteins together to treat hemophilia A and potentially extend the time between doses. The medication will allow patients to infuse once a week instead of twice-weekly for prophylactic treatment. See the full article at raredr.com.
Man With Severe Hemophilia Talks About Climbing Mount Everest
Chris Bombardier joins the list of Coloradans who have climbed Mount Everest, with a condition that makes it all the more impressive: he has severe hemophilia. Local Denver news station 9News interviews and talks to Chris about his achievement and what it means for those with bleeding disorders around the world. See video and article at 9news.com.
Iowa Teen's $1 Million-Per-Month Illness No Longer a Secret
Iowa's largest health insurer, Wellmark Blue Cross & Blue Shield, has cited a patient who has $1 million per month health bills as a reason for fast rising premiums of tens of thousands of other Iowans who buy their own insurance. The patient in question, it was revealed, has a severe bleeding disorder, hemophilia. See the full article at msn.com.
Sangamo Receives Fast Track Designation from FDA for SB-525 Gene Therapy
Sangamo Therapeutics, Inc. announced that the FDA has granted Fast Track designation to SB-525, the company's clinical stage cDNA gene therapy candidate for hemophilia A. The treatment is being developed as part of a collaborative relationship with Pfizer. See the full article at prnewswire.com.
FDA Approves Novo Nordisk's Rebinyn to Treat Hemophilia B
Novo Nordisk announced that the FDA has approved Rebinyn for the treatment of adults and children with hemophilia B. The new treatment is indicated for on-demand and perioperative management of bleeding, but not for routine prophylaxis. See the full article at prnewswire.com.
Climber from Colorado with Hemophilia Summits Mount Everest
Coloradan Chris Bombardier summits Mount Everest on May 22nd and becomes the first person with hemophilia to do so. Chris dedicated his climb to others with bleeding disorders and is spreading the word to raise support for saveonelife.net, a non-profit dedicated to helping those with bleeding disorders in developing countries. See the video from 9news here.
Dimension Therapeutics Discontinues Development of DTX101 Gene Therapy
Dimension Therapeutics reported their decision to discontinue the development of DTX101. The investigational AAVrh10 based gene therapy product was in development for the treatment of hemophilia B. The decision followed a review of the Phase 1/2 clinical study data that indicated the data would not meet their minimum target product profile for continued research. See the full article at pipelinereview.com.
Sangamo and Pfizer to Collaborate on Hemophilia A Gene Therapy
Sangamo and Pfizer have announced an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy treatments for hemophilia A, including SB-525. The partnership divides up financial, research, development, manufacturing and commercialization duties and allows the two to collaborate on future products. See the full article at pharmabiz.com.
FDA Grants Orphan Drug, Fast Track Designation to Two Hemophilia Products
Two gene therapies from Sangamo Therapeutics for hemophilia A and B have been granted special regulatory designations by the FDA. SB-525 to treat hemophilia A was granted orphan drug status and SB-FIX to treat hemophilia B was provided fast track designation. See the full article at hemophilianewstoday.com.
Prophylaxis Usage Analysis Authored by Dr. Marilyn Manco-Johnson Published
Dr. Marilyn Manco-Johnson, the Director of our HTC, is a primary author on a paper analyzing the use of prophylaxis over the past decade. The article, Prophylaxis usage, bleeding rates, and joint outcomes of hemophilia, 1999 to 2010: a surveillance project, was published in the April 27, 2017 edition of Blood, a journal produced by the American Society of Hematology. See the full article at bloodjournal.org.
Children's Hospital Colorado Highlights Dr. Jorge DiPaola
Children's Hospital Colorado highlighted our HTC's Dr. Jorge DiPaola who works with Cancer and Blood Disorders in a small video clip they shared on social media. He speaks about how he breaks the ice and connects with kids and teens. The video captures his great personality. See the video at childrenscolorado.org.
Man from Colorado with Hemophilia is Making His Way up Mt. Everest
Chris Bombardier, a mountaineer who grew up in Colorado and one of our patients at the HTC, is attempting to be the first person with severe hemophilia to attempt to summit Mt. Everest. He is sharing photos and updates at his Facebook page and is being documented by Believe Limited. His efforts are to bring awareness and support for saveonelife.net. A great article on this adventure can be found at purpose2play.com.