Data Supporting Long-Term Safety of Eloctate Published in Haemophilia
Newly published clinical data demonstrate that people on extended-interval prophylaxis regimens with Eloctate experienced low bleeding rates. Biogen and Swedish Orphan Biovitrum AB (Sobi) announced the results of the Phase 3 study called ASPIRE in Haemophilia, the journal of the World Federation of Hemophilia, the European Association for Haemophilia and Allied Disorders, and the Hemostasis & Thrombisis Research Society. See full article at marketwatch.com.
Hemophilia Drugs a Big Part of Medicaid Spending
Treatment costs for hemophilia appear to use up a large portion of a state's Medicaid budget, according to a study published in the July 28 issue of the Journal of the American Medical Association.
See full article at doctorslounge.com.
FDA Publishes Drug Shortage Ruling
The US FDA released a final rule on "Permanent Discontinuance or Interruption in Manufacturing of Certain Drug or Biological Products," which sets new rules to address drug shortages. The NHF has advocated for the inclusion of all biologics, including plasma-derived and recombinant products which are used by those in the bleeding disorder community. See full article at hemophilia.org.
Engineered Clotting Protein Effective in Severe von Willebrand Disease
The first protein engineered to help control bleeding episodes in patients with severe von Willebrand disease has been shown to be safe and effective, according to results of a Phase III trial. Study data were published in Blood, the Journal of the American Society of Hematology (ASH). See full article at hematology.org.
FDA Accepts CSL Behring's BLA for its rVIII-SingleChain Therapy for Hemophilia A
CSL Behring announced that the US FDA has accepted for review the company's Biologics License Application for its novel investigational recombinant factor VIII single-chain (rVIII-SingleChain) for the treatment of hemophilia A. The rVIII-SingleChain met all primary endpoints in a pivotal clinical trial. See full article at pipelinereview.com.
Bristol-Myers, AbbVie get FDA Approval for Hepatitis C Treatments
The US Food and Drug Administration approved two treatments for less common forms of hepatitis C infections. Bristol-Myers' Daklinza is used to treat Hepatitis C genotype 3 infections while AbbVie's Technivie targets the genotype 4 infections. See full article at reuters.com.
FDA Approves Technivie for Treatment of Chronic Hepatitis C Genotype 4
The US Food and Drug Administration has approved Technivie (ombitasvir, paritaprevir and ritonavir) for use in combination with ribavirin for the treatment of hepatitis C genotype 4 infections in patients without scarring and poor liver function. See full article at fda.gov.
Gene Sequence Swap Using CRISPR May Lead to Cure for Hemophilia A
For the first time, genetic defects responsible for hemophilia A have been corrected by using CRISPR-Cas9. Researchers at Yonsei University in South Korea were able to cure mice of hemophilia A with the precise procedure targeting only parts of the genome that they chose. See full article at news-medical.net.
Hepatitis Drug Overtakes $1000-per-pill Treatment in Shifting Options for Care
The new leading pill for hepatitis C is more expensive, and the number of patients seeking a cure has surged. Harvoni, also made by Gilead-the maker of the cure Sovaldi, has pushed aside it's predecessor despite the increased cost to $1350 per pill. The treatment is preferred for its simplicity. See full article at mercurynews.com.
CU Doctor Earns Awards To Research Improving Hemophilia Outcomes
Dr. Beth Warren, a Fellow at the University of Colorado Anschutz Medical Campus, working with Children's Hospital Colorado and the Hemophilia and Thrombosis Center, has earned two awards to provide the ability to further research in improving outcomes for hemophilia patients. See full article at DenverPost.com.
Baxalta Launches as Spinoff of Baxter Aimed at Underserved Conditions
Baxalta Incorporated launched on July 1st as a global biopharmaceutical company, spinning off from Baxter. Baxalta will manage the hematology research and products designed to serve those with hemophilia and other bleeding disorders. See full article at businesswire.com.
Bayer Files Hemophilia A Drug Marketing Application in Japan
Bayer recently announced they have submitted a Marketing Authorization Application in Japan for BAY 81-8973, a recombinant factor VIII compound. The company has already submitted regulatory applications for the treatment in the US and EU for the treatment of hemophilia A. See full article at zacks.com.
Walgreen Settles Medicaid False Billing Case in New York
Walgreen Co will pay $2.55 million to settle charges by New York's attorney general that a pharmacy unit improperly billed Medicaid for costly drugs to treat hemophilia patients without proof of delivery. Walgreen settled to avoid delay, expense, and litigation but did not admit liability. See full article at reuters.com.
New Data Presented on the Impacts of Hemophilia on Caregivers and Patients
Novo Nordisk presented data on two separate studies investigating caregivers and patients that showed many caregivers experience significant emotional stress and concern, and indicated a high prevalence of acute or chronic pain among hemophilia patients. See full article at news-medical.net.
CSL Behring Presents Data from Phase I/III rVIII Single-Chain Study
CSL Behring presented data from a Phase I/III study on its recombinant factor VIII for the treatment of Hemophilia A in both adolescents and adults. The results were presented at the International Society on Thrombosis and Haemostasis Congress in Toronto June 20-25th. See full article at news-medical.net.
Novo Nordisk Presents Positive Data for the Treatment of Hemophilia A
At the International Society on Thrombosis and Haemostasis Congress in Toronto, Novo Nordisk presented positive data for the treatment of hemophilia A from the Phase 3 trial of Zonovate (known as NovoEight in the US). See full article at newswire.ca.
Baxalta Provides Update on Progress with Hemophilia B Gene Therapy
Baxalta, a new independent Baxter drug spinoff research company, recently provided early results from a Phase I/II study of their long-term gene therapy for hemophilia. Researchers say they have recorded initial responses in their dose escalation study of BAX 335 for hemophilia B. See full article at fiercebiotech.com.
Catalyst Biosciences Announces Positive Results from Phase I Trial for CB 813d to treat Hemophilia
Catalyst Biosciences announced positive results from a Phase I clinical trial for it's next-generation and long-acting coagulation factor VIIa treatment CB 813d. Results were presented at the International Society on Thrombosis and Haemostasis Congress in Toronto in June. See full article at globenewswire.com.
CSL Behring Presents rIX-FP Data for Phase III trial to treat Hemophilia B
CSL Behring presented data at the International Society on Thrombosis and Haemostasis Congress in Toronto from Phase III studies evaluating the safety of its long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP). See full article at prnewswire.com.
Alnylam Reports Positive Data for ALN-AT3 for Treatment of Hemophilia A & B
Alnylam Pharmaceuticals announced new positive data from its ongoing Phase 1 clinical trial with ALN-AT3 for hemophilia treatment at the International Society on Thrombosis and Haemostasis 2015 Congress held on June 20-25 in Toronto, Canada. See full article at pharmiweb.com.
Quick Action Saves Teen from Stroke
One of our patients, Autumn Voldrich, was recently interviewed at our center by our local CBS station in Denver. Autumn has worked with Dr. Armstrong-Wells since her stroke two years ago and has made beautiful progress, much of it because of the quick response of her parents and an astute paramedic. See full article and video at cbsnews.com.
Apitope's Inhibitor Drug Candidate ATX-F8-117 Granted FDA Orphan Drug Status
The drug development company Apitope announced that their pre-clinical product candidate ATX-F8-117 has been granted Orphan Drug Status by the FDA for the prevention or treatment of inhibitors in hemophilia A patients who have inhibitors or are at risk of producing inhibitors. See full article at pharmabiz.com.
Mountaineer and Hemophiliac Chris Bombardier to Conquer Highest Summit on Each Continent
Chris Bombardier is on a quest to climb the Seven Summits, the highest peaks on every continent. He hopes to raise awareness about hemophilia and raise funds to help young hemophiliacs in developing countries through Save One Life. Chris grew up in Colorado and not only used our HTC for his medical needs, but worked at our center. See full article at purpose2play.com.
FDA Approves IXINITY from Emergent BioSolutions to Treat Hemophilia B
Emergent BioSolutions announced that the US Food and Drug Administration has approved IXINITY, an intravenous recombinant human coagulation factor IX therapeutic for the control and prevention of bleeding episodes for those with hemophilia B. See full article at emergentbiosolutions.com.