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.
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.