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.
Baxalta Submits sBLA for Adynovate to Treat Pediatric Hemophilia A Patients
Baxalta announced that it has submitted supplemental Biologics License Applications (sBLA) to the FDA seeking approval for the use of Adynovate to treat children under the age of 12 with hemophilia A and for use in surgical settings. See full article at pharmabiz.com.
NHF Provides Updates on Actions Being Taken to Keep Blood Supply Safe
The National Hemophilia Foundation has been working closely with the CDC, the WFH and other industry organizations to monitor the spread of the Zika virus. They have posted information as well as statements by producers of plasma products such as the PPTA and the AABB on how they are working to keep the blood supply safe from this and other viruses. See full article at hemophilia.org.
WFH Releases Statement on Zika Virus and Plasma-Derived Treatment Products
The World Health Organization (WHO) has declared the Zika virus a public health emergency of international concern. The World Federation of Hemophilia has posted an article with history and details about the virus, its history, and how they are monitoring the possible spread to clotting factor concentrates and any risk to bleeding disorder patients. See full article at wfh.org.
Red Cross Takes Preventative Steps to Prevent Zika Virus in Blood Supply
The American Red Cross is asking potential blood donors who have traveled to areas where the Zika infection is active to wait 28 days before giving blood. These precautionary measures are similar to those for other illnesses and include screening processes to only collect blood from donors who are healthy. See full article at MedlinePlus from the NIH.
HFA Provides Updates and Info on Zika Virus and the Blood Supply
The Hemophilia Federation of America posted updates and links to provide information to the public about the possibility of the Zika Virus in the blood supply. Of most concern, and updates are provided, are the safety of plasma-derived products that can be used in bleeding disorder medications. See full article at hemophiliafed.org.
Gene Therapy Research Shows Potential for Long Lasting Treatment
Researchers have used a single injection of gene therapy to correct a rare bleeding disorder, factor VII deficiency, in dogs. This success in large animals holds considerable potential for a safe, effective and long lasting new treatment in humans with the same bleeding disorder. See full article at eurekalert.org.
FDA Approves Zepatier to Treat Chronic Hepatitis C Genotypes 1 and 4
The US Food and Drug Administration approved Zepatier (elbasvir and grazoprevir) with or without ribavirin for the treatment of chronic hepatitis C virus (HCV) genotypes 1 and 4 infections in adult patients. This provides another oral treatment option for patients without the use of interferon. See full article at fda.gov.
Gilead Faces Scrutiny, Lawsuits Over Pricing of Treatments for AIDS/Hep C
Gilead Sciences continues to face scrutiny over the pricing of some of its drug treatments. The company was threatened with a lawsuit addressing the pricing of their successful hepatitis C treatments Harvoni and Sovaldi. In addition, the company is facing challenges to patents it holds on its AIDS drug, Tenofovir. See full article at biospace.com.
March Officially Named Bleeding Disorders Awareness Month
The US Department of Health and Human Services (HHS) has approved "Bleeding Disorders Awareness Month" to be included in their National Health Observances calendar beginning in 2016. This formalizes and expands upon the designation given 30 years ago when "Hemophilia Awareness Month" was announced by President Ronald Reagan. See full article at hemophilia.org.
Hemophilia Patients' Dual Roles Creates Ethical Quandary
Drugs for hemophilia are so expensive that it has become lucrative for the pharmaceutical industry to blur the lines between being patients and drug sellers. Manufacturers of hemophilia drugs and specialty pharmacies that dispense them are hiring patients and their relatives to gain an inside track and access in selling products. It has led to an ethical gray area. See full article at nytimes.com.
Drug Maker Shire to buy Baxalta for $32 Billion
Ireland based Shire has managed to strike a deal to purchase Baxalta, the US Baxter spinoff that produces treatments for rare blood conditions, cancers, and immune system disorders. The move has potentially pushed Baxalta into a leading position in treating rare diseases, including hemophilia. See full article at reuters.com.
Gilead Snags Priority Review for Pangenotype Hepatitis C Combo Treatment
The US Food and Drug Administration has granted a priority review for Gilead's fixed-dose combination of Sovaldi and velpatasvir, designed to treat genotypes 1-6 of the hepatitis C virus. Sovaldi is currently approved for genotypes 1-4. The combination treatment would expand the number of treatable genotypes. See full article at biopharmadive.com.
Octapharma's NUWIQ Now Available in the US to Treat Hemophilia A
Octapharma USA announced that their new Antihemophilic Factor (Recombinant), marketed as NUWIQ, is now commercially available in the United States to treat Hemophilia A. NUWIQ is indicated for use in both children and adults for routine prophylaxis, treatment of bleeds, and perioperative management. See full article at businesswire.com.
Sernova Awarded Grant to Fund Development of Cell-Based Treatment for Hemophilia A
Sernova Corp. announced that the European Commission's Horizon 2020 program has awarded a $8.5 grant to a consortium consisting of Sernova and five European academic and private partners to advance development of a GMP clinical grade Factor VIII releasing therapeutic cell product in combination with Sernova's Cell Pouch to treat Hemophilia A. See full article at sernova.com.
SIPPET Study Results Presented at ASH 2015
The results of the SIPPET (Survey of Inhibitors in Plasma-Products Exposed Toddlers) study were presented at a plenary session of the recent American Society of Hematology (ASH) conference held in Orlando. The study collected data on 251 children under six years of age with severe hemophilia A from 14 countries. See full article at hemophilia.org.
Arsia & Biogen Unite to Develop Subcutaneous Method for Delivering Hemophilia Medications
Two-year old startup Arsia Therapeutics Inc. announced that it will collaborate with biotech giant Biogen Inc. to develop a system for delivering drugs to hemophilia patients through under-the-skin injections. Arsia's delivery technology seeks to reformulate drugs that can now be delivered only via intravenous infusion, making them more convenient. See full article at bostonglobe.com.
NHF CO Selects Denver Brandworks to Expand Backpacks and Bleeders
The Colorado Chapter of the National Hemophilia Foundation (NHF Colorado) has engaged Denver Brandworks to design and implement a national expansion strategy for one of its most successful community programs, Backpacks & Bleeders. Because of its local success, sponsor Beyer has asked NHF Colorado to steward expansion of the program nationally. See full article at pr.com.
FDA Approves Vonvendi as First Recombinant von Willebrand Factor Treatment
The US Food and Drug Administration has approved Vonvendi, von Willebrand factor (Recombinant), for use in adults 18 years of age and older who have von Willebrand disease (VWD). Vonvendi is the first FDA-approved recombinant von Willebrand factor and is approved for on-demand and episodic bleeds for those with the disorder. See full article at fda.gov.
New Data Shows Eloctate & Alprolix May Manage Target Joint Bleeds and Maintain Low Annual Bleeding Rates
New data from Biogen and Sobi on their Eloctate (factor VIII, Fc fusion protein) and Alprolix (rFIXFc) indicates that the medications may effectively manage target joint bleeding and maintain low annualized bleeding rates in people with severe hemophilia A and B. The data was presented at the 2015 American Society of Hematology Annual Meeting held in Orlando, FL. See full article at pharmiweb.com.
Positive Data For Alnylam's Once-Monthly Fitusiran (ALN-AT3) to Treat Hemophilia A & B
Alnylam Pharmaceuticals, Inc. announced positive results from its ongoing Phase I clinical study with fitusiran (ALN-AT3), a treatment designed to lower levels of antithrombin (AT) for the treatment of hemophilia A and B. The data were presented at the American Society of Hematology (ASH) 2015 Annual Meeting in Orlando, Florida. See full article at businesswire.com.
New Data Presented by Sangamo BioSciences on SB-FIX, Clotting Protein to Treat Hemophilia B
Sangamo BioSciences, Inc., which is working in genome editing to address hemophilia B, shared data from recent research from its ZFP Therapeutic program at the 57th Annual American Society of Hematology Meeting in Orlando, FL. Their data supports further research in a forthcoming clinical trial of SB-FIX which would be the first clinical application of their proprietary In Vivo Protein Replacement Platform. See full article at prnewswire.com
Baxalta Shares Updates on Trial of ADYNOVATE to Treat Hemophilia A
Baxalta Inc. showcased it's most recent data from trials of their recently FDA approved ADYNOVATE, a new treatment for hemophilia A at the 57th Annual American Society of Hematology Meeting (ASH) in Orlando, FL. The trials studied prophylactic treatment as well as joint bleeding patterns, and perioperative control. See full article at streetinsider.com.
CSL Behring Presents Data From Phase III Trial for Long Acting rIX-FP to Treat Hemophilia B
Data presented by CSL Behring at the 57th American Society of Hematology's Annual Meeting (ASH) in Orlando, Florida confirm earlier findings about the safety and efficacy of their rIX-FP to treat hemophilia B. Patients in the study extended their prophylaxis treatment to dosing intervals of up to 14 days. See full article at prnewswire.com.
FDA Accepts New Drug Application for AbbVie's Once-a-day VIEKIRA PAK to Treat Hepatitis C Genotype 1
The US Food and Drug Administration has accepted the New Drug Application for VIEKIRA PAK, an all-oral, interferon-free treatment as a once-daily dose for those with genotype 1 chronic hepatitis C infection. AbbVie already has a similar product that is taken twice daily. See full article at prnewswire.com.
FDA Clears Investigational New Drug Application by Sangamo BioSciences for SB-FIX to Treat Hemophilia B
The US Food and Drug Administration has cleared Sangamo BioSciences' Investigational New Drug application for SB-FIX. This potentially curative, single treatment therapy for hemophilia B is the first in vivo genome editing application to go to clinical trials and first human study of its kind. See full article at prnewswire.com.
Study of ACE910 Suggests Treatment May Result in Fewer Injections to Treat Hemophilia A
An antibody engineered to prevent excessive bleeding in patients with severe hemophilia A may be safe and effective, and require fewer injections than existing options, according to a first-in-human study of the treatment published online in Blood, the Journal of the American Society of Hematology (ASH). See full article at hematology.org.