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.
FDA Approves Adynovate for Hemophilia A
The US Food and Drug Administration has approved Adynovate, Antihemophilic Factor (Recombinant), PEGylated for use in adults and adolescents, aged 12 years and older, who have Hemophilia A. Adynovate is modified to last longer in the blood and potentially require fewer infusions than unmodified factor. See full article at fda.gov.
BDI Pharma, Inc. Selected as Authorized Distributor of Coagadex
BDI Pharma, Inc. has been selected as the authorized distributor of record for Coagadex, the newly approved treatment for adults and children with hereditary factor X deficiency. The medication will provide on-demand treatment and control of bleeding episodes, and perioperative management of bleeding. See full article at prnewswire.com.
Research at Rice University Yields New Answers About Factor VIII
Researchers at Rice University have solved a long-standing mystery about where the body stores and deploys blood-clotting factor VIII, a protein that about 80 percent of those with hemophilia cannot produce due to genetic defects. See full article at news.rice.edu.