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.
FDA Approves First Factor X Concentrate To Treat Patients with Bleeding Disorder
The US Food and Drug Administration approved Coagadex, Coagulation Factor X (human) for hereditary Factor X deficiency. Until this most recent approval, no specific coagulation factor replacement therapy was available for patients with hereditary Factor X deficiency. See full article at fda.gov.
Lettuce Can Make Drugs That Don't Require Refrigeration
The University of Pennsylvania School of Dental Medicine researchers have been working to create biopharmaceuticals, such as insulin for diabetics or factor for those with bleeding disorders, in an affordable and shelf-stable way. They have used freeze-dried lettuce as a plant based method to deliver these protein based products and reduced inhibitors in mice. See full article at futurity.org.
Alnylam Pharma Announces Phase 1 Study of ALN-AT3 for Hemophilia
Alnylam Pharma announced that it has initiated a Phase 1 open-label extension (OLE) study with ALN-AT3, an investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia. This study will evaluate the long- term safety and efficacy of ALN-AT3. See full article at streetinsider.com.
Octapharma Licenses Glycotope Platform; Takes Stake in Company
Octapharma announced that it has taken a minority stake in Glycotope and acquired an exclusive worldwide license to its IP covering its recombinant technology. In addition, Octapharma said it will join Glycotope on a series of R&D projects to enable the technology transfer. See full article at genengnews.com.
New Hepatitis C Combination Shows Positive Results Across All Genotypes
Gilead reported positive data from four late-stage studies showing that a combination featuring its Sovaldi and the experimental NS5A inhibitor velpatasvir is effective across all hepatitis C virus genotypes. The finding is potentially important because it could eliminate the need for genotype testing prior to treatment. See full article at hcplive.com.
Study Shows Infections May Temporarily Increase Children's Risk of Stroke
A study published in Neurology found that the flu and colds may temporarily increase the risk of stroke in children, and that vaccines may decrease the risk of stroke. Research was done by Heather J. Fullerton of UCSF Benioff Children's Hospital in San Francisco and studied children diagnosed with stroke. Several of those involved in the study were treated at our center. See full article at biosciencetechnology.com.
BioMarin Enrolls Patients in Gene Therapy Trial for BMN 270 to Treat Hemophilia A
BioMarin Pharmaceutical Inc. announced that it has enrolled it's first patients in a phase 1/2 trial for BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A. BMN 270 is an AAV-Factor VIII vector designed to restore factor VIII plasma concentrations. See full article at globenewswire.com.
NHF Announces Comprehensive Care Sustainability Collaborative (CCSC)
The National Hemophilia Foundation (NHF) and Impact Education, LLC. recently announced the Comprehensive Care Sustainability Collaborative, an ongoing quality improvement and cost-management initiative utilizing the insights of a group of HTC directors, clinicians and administrators working with payer/managed care and pharmacy directors to improve cost-effective hemophilia management. See full article at hemophilia.org.
FDA Grants Fast Track Designation for Dimension's DTX101 to Treat Hemophilia B
Dimension Therapeutics, Inc. announced that the FDA has granted Fast Track designation for DTX101, to treat hemophilia B. The company expects to initiate a multi-center phase 1/2 study to evaluate their product in adult patients by the end of 2015. See full article at dimensiontx.com.
FDA Approves Octapharma's NUWIQ to Treat Hemophilia A
The FDA has approved Octapharma's NUWIQ, antihemophilic factor (recombinant), therapy for those with hemophilia A. This includes on-demand and routine prophylaxis therapies. NUWIQ is the first B-domain-deleted factor VIII derived from a human cell line, not chemically modified or fused with another protein that is designed for treating hemophilia A. See full article at pharmacypracticenews.com
FDA Accepts Dimension's DTX101 as Investigational New Drug and Grants Orphan Designation to Treat Hemophilia B
The FDA has accepted Dimension's investigational new drug application and granted orphan drug designation for the company's lead product candidate, DTX101, for the treatment of hemophilia B. Dimension expects to initiate a multi-center phase 1/2 study in adults to evaluate DTX101 with moderate to severe hemophilia B by the end of 2015. See full article at businesswire.com.
FDA Grants Breakthrough Therapy Designation to Genentech's ACE910 to treat Hemophilia A with Factor VIII Inhibitors
Genentech announced that the US FDA has granted breakthrough therapy designation to ACE910 for the treatment of patients with hemophilia A with factor VIII inhibitors. This designation is designed to accelerate the development and review of medicines that demonstrate evidence of a substantial improvement over current treatment options. See full article at businesswire.com.
World Leaders Meet at First World Hepatitis Summit to Discuss Prevention
The World Health Organization (WHO) and the World Hepatitis Alliance (WHA) organized the first ever World Hepatitis Summit held in Glasgow, Scotland. The three day meeting addressed the growing rise in deaths worldwide due to viral hepatitis, which has become the 7th biggest annual killer worldwide. See full article at news-medical.net.
Recombinant FVIII with Extended Half-Life Shown Safe, Effective for Hemophilia A
In a phase 2/phase 3 study by Baxalta, their BAX 855, a pegylated, full-length recombinant Factor VIII, appeared safe and effective for the biweekly prophylactic treatment of patients with hemophilia A. The study indicates potential for additional therapeutic options for patients to reduce the frequency of prophylactic infusions. See full article at healio.com.
CSL Behring Enrolling for Study of rVIIa-FP for Hemophilia Patients with Inhibitors
CSL Behring announced that they have enrolled the first of approximately 54 male patients for their Phase II/III clinical study evaluating the pharmacokinetics, efficacy, and safety of their rVIIa-FP for on-demand treatment in patients with hemophilia A or B who have developed an inhibitor to factor VIII or factor IX replacement therapy. See full article at news-medical.net.
Biogen Issues Positive Update on Alprolix Phase III Study for Hemophilia B
New clinical data supporting the long-term safety and efficacy of ALPROLIX [Coagulation Factor IX (Recombinant), Fc Fusion Protein] in people with severe hemophilia B showed patients maintained low bleeding rates with one to two week prophylaxis regimens. See full article at streetinsider.com.
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.