Roche Reports Encouraging Data for Emicizumab at WFH Congress
Roche Reported positive data for their top candidate emicizumab (formerly ACE910) at the 2016 World Federation of Hemophilia World Congress in Orlando. The drug cut the bleeding rate of patients by more than 95% after a follow up review lasting 32.6 months from a small study. See full article at endpts.com.
BioMarin Shares Dramatic Results from Gene Therapy Research
BioMarin released data at the WFH World Congress in Orlando, Florida showing dramatically improved factor levels from their small study of gene therapy patients. The treatment produced results for hemophilia A patients showing factor levels within the normal range with minimal toxicity issues. See full article at forbes.com.
Sangamo BioSciences Reveals New Gene Therapy Development Program
Sangamo BioSciences presented preclinical data supporting the clinical development of its new proprietary gene therapy program for hemophilia A. The data from mouse and monkey subjects was presented at the 2016 WFH World Congress held in Orlando, Florida. See full article at fiercebiotech.com.
CSL Behring Presents Data for Afstyla in Adolescents and Children
CSL Behring presented data from a Phase III pivotal study of Afstyla in hemophilia A patients 12 years old or younger at the 2016 WFH World Congress in Orlando. The results showed that Afstyla, when used prophylactically, showed low spontaneous bleeds in previously treated children. See full article at prnewswire.com.
Novo Nordisk Shares Data of Psychosocial Impact on People with Hemophilia B
Novo Nordisk announced their results from the Bridging Hemophilia B Experiences, Results and Opportunities into Solutions study (B-HERO-S), which found that many adults and children with hemophilia B, even in mild cases, experience a negative impact in their lives due to their disorder. See full article at prnewswire.com.
CSL Behring Presents Phase III Data for Idelvion to Treat Hemophilia B
CSL Behring presented new data from its Phase III PROLONG-9FP clinical development program evaluating Idelvion at the 2016 WFH World Congress in Orlando. These new data show long-term safety and efficacy in prophylaxis over time in pediatric and adult patients with hemophilia B. See full article at cslbehring.com.
Alnylam Pharma Announces Positive Data for Fitusiran Phase I Study
Alnylam Pharma announced at the 2016 World Congress in Orlando new positive results from it's ongoing Phase 1 study with fitusiran, an investigational RNAi therapeutic targeting antithrombin for the treatment of Hemophilia A and B and rare bleeding disorders. See full article at streetinsider.com.
CSL Behring Announces Voluntary Recall of Two Lots of Helixate FS
CSL Behring has announced a voluntary recall of two lots of Helixate FS with vial adapter. The recall is based on stability testing that revealed a loss of potency to below pre-specified acceptable levels. The recall is not an issue of safety of the product. For lot numbers involved and more information go to hemophilia.org.
Bayer Announces Voluntary Recall of Two Lots of Kogenate FS
Beyer has announced a voluntary recall of two lots of Kogenate FS with vial adapter. The recall is based on stability testing that revealed a loss of potency to below pre-specified acceptable levels. The recall is not an issue of safety of the product. For lot numbers involved and more information go to hemophilia.org.
Spark Therapeutics and Pfizer Gain FDA Breakthrough Designation for SPK-9001
Spark Therapeutics and Pfizer announced that the FDA has granted breakthrough therapy designation to SPK-9001 to treat hemophilia B. SPK-9001 is in an ongoing Phase 1/2 trial as a potential one time therapy by using a novel bio-engineered adeno-associated virus (AAV). See full article at streetinsider.com.
New Podcast for Bleeding Disorders Launches
A new podcast that provides news, updates on medications, interviews and opinion about issues affecting those with bleeding disorders launched in June. The host, Patrick James Lynch, is a well known community member and advocate for those with bleeding disorders. For more information go to bloodstreampod.com.
Koate DVI Shipments on Hold
Kedrion Biopharma announced it was placing a voluntary, temporary hold on future shipments of Koate DVI, a treatment for hemophilia. It is anticipated that no new shipments will begin until September 2016. Please contact your physicians for alternate treatments or assistance in managing care if you use this medication. See full article at hemophilia.org.
FDA Approves Epclusa to Treat Hepatitis C
The US Food and Drug Administration has approved Epclusa to treat adult patients with chronic hepatitis C virus both with and without cirrhosis. The approval offers additional treatment options for a wider scope of patients with hepatitis C. Epclusa is manufactured and marketed by Gilead. See full article at fda.gov.
SIPPET Study & MASAC Recommendations Explained
Patrick James Lynch, a hemophilia patient and public advocate for those with bleeding disorders, posted an article at the request of the NHF to help explain issues and analyze the recommendations that have arisen after the publication of the SIPPET Study (Survey of Inhibitors in Plasma-Products-Exposed Toddlers). See full article at bloodstreampod.com.
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.