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.
Thousands Reach Out After Perth Woman's Bleeding Disorder Story
Chloe Christos shared her story last year about bleeding five years straight after she started her first period to help bring awareness to bleeding disorders. Since her story about living with von Willebrand Disease went public, thousands of friends and strangers have contacted her. Her story and the many others who contacted her, have prompted her to create an online platform to connect. See full article at abc.net.au.
Making Strides Toward Preventing Inhibitors in Bleeding Disorders
The CDC has updated their informational webpage that discusses how the CDC is monitoring inhibitors and ways specialists and researchers are working to manage and prevent inhibitors. The Second Inhibitor Summit and the plans that the CDC has in place to develop methods to test and learn about inhibitors are outlined. See full article at cdc.gov.
Roche Reports Positive Results for Phase III HAVEN 2 Study
Roche has announced interim results from the phase III Haven 2 study evaluating emicizumab prophylaxis in children less than 12 years old with hemophilia A and inhibitors to Factor VIII. This analysis showed a clinically meaningful reduction in the number of bleeds over time. See full article at pharmabiz.com.
Catalyst Biosciences Reaches Milestone to Advance Factor IX Program
Catalyst Biosciences announced that it has achieved a key milestone to advance the Factor IX program towards its first human clinical trial. The highly potent Factor IX variant has demonstrated potential to normalize human Factor IX levels with a daily subcutaneous injection in preclinical studies. See full article at globenewswire.com.
Ryan White's Letters to go on Display
Ryan White, the teenager with hemophilia who gained international attention when he contracted the AIDS virus from contaminated blood, received thousands of letters from children during his life. A new project between his mother and the Children's Museum of Indianapolis is preparing the letters to put on display and offer insight on how children viewed the AIDS epidemic. See full article at cbs4indy.com.
Aptevo Therapeutics Announces New IXINITY Supply Available May 2017
Aptevo Therapeutics announced a successful manufacture of a recent bulk drug substance (BDS) batch of IXINITY. The medication is an intravenous recombinant factor IX therapeutic for use in people with hemophilia B. The new supply will be available beginning in May 2017. See full article at globenewswire.com.
Amy Board of NHF and Chris Bombardier Interviewed on Colorado & Company
Amy Board of NHF Colorado and local Chris Bombardier were interviewed on Denver's local NBC station 9News on "Colorado & Company" a local morning show. The video was an opportunity to share several NHF programs such as Easy Brews, Backpacks & Bleeders, as well as discuss Chris Bombardier's goal of hiking the Seven Summits, including Mt. Everest-which he will attempt this Spring. See video clip at youtube.com.
Washington Days Brings Big Crowd to Capitol Hill
The National Hemophilia Foundation hosted its annual Washington Days advocacy event on March 8-10, 2017 in Washington DC. A record number of participants attended this year, with more than 480 volunteer advocates from 46 states. These members of local communities met with their legislators to discuss the importance of medical protections needed by those with bleeding disorders. See full article at hemophilia.org.
CU Anschutz Medical Campus Bus Shuttle a Transit Success
CU Anschutz Medical Campus is now offering a free shuttle bus that allows RTD riders of the newly launched light rail R line easy access all over the Anschutz medical campus. The blue busses have several drop off and pick up points around the campus, including one that is just south of the UPI building where the HTC is located on Montview Boulevard. See full article at cuanschutztoday.org.
"My Life, Our Future" Repository Opens Data to Researchers
The research repository created by the collection of genetic information and blood samples from more than 5000 hemophilia patients in the US is being made available to researchers. The "My Life, Our Future" project collected the data to help scientists make advancements in hemophilia research. Scientists must submit a proposal for evaluation in order to use the data. See full article at raredr.com.
Chris Bombardier Takes on Mount Everest
Chris Bombardier, a member of the Colorado bleeding disorders community, will be attempting to hike Mount Everest in April 2017. He strives to bring awareness and support families with hemophilia in countries without the care available in the US, such as Nepal. If successful, he will have six out of the seven peaks finished with Mount Everest. See full article at saveonelife.net.
Data from Long Term Study of Alprolix to Treat Hemophilia B Published
Bioverativ, a recent spin off from Biogen, and Sobi published data from a long term study of Alprolix, a recombinant clotting factor therapy for hemophilia B. The B-YOND extension study confirm the long-term safety and efficacy of Alprolix, and revealed that a majority of participants were able to dose once weekly or less. See full article at businesswire.com.