Analysis Published in Blood Reviews Outcomes
In an article published in Blood in June, authors studied data from nearly 5000 men in the United States with hemophilia and found an ongoing need of care. The HTC's Brenda K. Riske was co-author on the study which now includes additional commentary as part of the report. See full article at bloodjournal.org.
Drug Makers Work to Woo Kids With Hemophilia
To reach the small, but lucrative hemophilia market, some drug makers have tried an unusual and high-intensity promotional strategy- building lifetime relationships. In a paper published in PLOS Medicine, researchers showed that methods used by pharmaceutical companies may undermine effective decision-making and likely requires more oversight. See full article at statnews.com.
Spark Therapeutics Releases Positive Data on Gene Therapy
Spark Therapeutics announced updated results of the first cohort from the ongoing Phase I/II clinical trial of their SPK-9001 to treat hemophilia B. All four subjects received a one-time administration of the therapy and experienced consistent and sustained factor IX activity levels. See full article at ir.sparktx.com.
uniQure Presents Data From Gene Therapy Trial for Hemophilia B
The gene therapy research company uniQure released additional data from their ongoing Phase I/II clinical trial of AMT-060 in hemophilia B patients at the 21st Congress of the European Hematology Association in Copenhagen, Denmark. Findings showed improvements over the six month post-treatment period for the five patient study. See full article at pipelinereview.com.
FDA Approves CSL Behring's Afstyla to Treat Hemophilia A
The US Food and Drug Administration has approved Afstyla, CSL Behring's long-lasting recombinant factor VIII single-chain therapy for adults and children with hemophilia A. Afstyla is indicated for use as routine prophylaxis to reduce the frequency of bleeding episodes, on-demand bleeding, and perioperative management of bleeding. See full article at pharmabiz.com.
SIPPET Study Results Published in New England Journal of Medicine
Detailed findings from the SIPPET (Survey of Inhibitors in Plasma-Products Exposed Toddlers) study were published in the New England Journal of Medicine. Marilyn Manco-Johnson, Director of our HTC, was a co-author on the study that focused on one of the most challenging complications for hemophilia patients globally. See full article at hemophilia.org.
US Health Officials Brace for Battle with Zika
US public health officials have begun deploying a three-pronged battle plan to combat the spread of Zika virus in the United States. They are working with federal, state and local resources to improve mosquito control, expand their ability to test for Zika, and launching campaigns to urge the public to protect themselves against mosquitoes. See full article at healthfinder.gov.
CSL Behring's Idelvion Receives Marketing Exclusivity From FDA
The US Food and Drug Administration granted seven years marketing exclusivity to CSL Behring's Idelvion, their medication approved in March to treat children and adults with hemophilia B. Idelvion is the first and only factor IX albumin fusion therapy that delivers extended dosing for hemophilia B patients. See full article at pharmabiz.com.
Biogen Announces Intent to Spin off its Hemophilia Business
Biogen Inc. announced that it intends to spin off its hemophilia business as an independent, publicly traded company. The new company has not yet been named and will focus on the discovery and development of therapies for the treatment of hemophilia, including its existing brands ELOCTATE and APROLIX. See full article at media.biogen.com.
Woman Fights For Females Living With Bleeding Disorders
A woman from Australia was 14 years old when she got her first period and didn't stop bleeding for five years. After a diagnosis of von Willebrand Disease, she is now fighting for the rights of women living with bleeding disorders around the world. See full article at abc.net.au.
The US Dept. of Health and Human Services Supporting Study of Zika Virus Blood Screening Test
The US Department of Health and Human Services is supporting the development of a test to identify whether donated blood is infected with the Zika virus. The screening could help ensure that infected blood is removed from the blood supply, keeping those using blood products from infection. See full article at hhs.gov.
BioMarin Provides Encouraging Update in Hemophilia A Gene Therapy Program
BioMarin Pharmaceutical Inc. announced preliminary data from an ongoing Phase 1/2 clinical trial of their BMN 270, an investigational gene therapy treatment for Hemophilia A. The small group of patients in the trial experienced improved factor levels, leading to the potential to eliminate the need for infusions in the future. See full article at rttnews.com.
Biogen Lights Up Major Structures, Raises Funds, and Donates Medications in Honor of World Hemophilia Day
Biogen announced they would join the World Federation of Hemophilia and local advocacy organizations to drive a number of activities across the US with the aim of raising awareness and encouraging support for World Hemophilia Day. They collaborated with groups to light up major structures across the world as well as donating medication and money. See full article at businesswire.com.
CSL Behring Marks World Hemophilia Day by Donating Medications for Bleeding Disorders to Developing Countries
CSL Behring announced that they have shipped more than 1.5 million international units of its specialty biotherapeutics to treat hemophilia A and/or von Willebrand Disease to the WFH Global Alliance program. Their gift to provide these much needed medications to those with bleeding disorders around the world was to support World Hemophilia Day. See full article at prnewswire.com.
Youth is Served in Stroke Save
A young man from Fort Lupton, Colorado playing baseball suddenly began having trouble speaking and fell. His coach called 911. With coordinated efforts by paramedics, Platte Valley Hospital, University of Colorado Hospital and Children's Hospital Colorado, including care from our HTC's Dr. Timothy Bernard, he is making a good recovery from a stroke. See full article at uchealth.org.
Research on Mice Shows Concurrent Influenza Vaccination Reduces Inhibitors
A study published in Blood indicates that vaccination against influenza decreases the risk of inhibitor development in hemophilia A mice. The study sought to test whether inflammatory signals created during immunization could be a risk factor in the development of inhibitors. The results showed vaccination against influenza does not increase the risk of inhibitor formation. See full article at bloodjournal.org.
Study of Hemophilia Care Reveals Progress, Ongoing Health Needs
Brenda Riske, Regional Coordinator for the Mountain States Hemophilia Region and Program Director at our HTC, co-authored a study which was published in the March 2016 issue of Blood. The study analyzed data of men with severe hemophilia and found improvements in care and treatments, but evidence of ongoing health disparities See full article at hematology.org.
FDA Approves Idelvion to Treat Hemophilia B
The US Food and Drug Administration approved Idelvion, a Factor IX (recombinant), albumin fusion protein, to treat patients with hemophilia B. The new agent is indicated for on-demand use for prevention and control of bleeding as well as perioperative bleeding management. See full article at healio.com.
Dr. Holbrook Khort, Hemophiliac Who Made Condition a Crusade, Dies at 38
Dr. Holbrook Khort, a researcher and medical doctor who worked in immune oncology at Stanford University has died from complications arising from hemophilia. His experience as a patient drove him to work in medical research to strive to find cures for disease. See full article at nytimes.com.
BioMarin Receives Orphan Drug Status from FDA for First AAV-Factor VIII Gene Therapy, BMN 270, to Treat Hemophilia A
BioMarin Pharmaceutical Inc. announced that their BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A, has been granted orphan drug designation by the FDA. BioMarin is conducting a phase 1/2 study to evaluate the safety and efficacy of the gene therapy. See full article at globenewswire.com.