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Hemophilia A is an X-linked, recessive, bleeding disorder caused by a deficiency in the activity of coagulation factor VIII. Affected individuals develop a variable phenotype of hemorrhage into joints and muscles, easy bruising, and prolonged bleeding from wounds. The disorder is caused by heterogeneous mutations in the factor VIII gene which maps to Xq28.
Apitope's ATX-F8-117 granted FDA Orphan Drug Designation for treatment of haemophilia A patients

Apitope's ATX-F8-117 granted FDA Orphan Drug Designation for treatment of haemophilia A patients

Apitope, the drug discovery and development company focused on disease-modifying treatments that reinstate immune tolerance, announced today that pre-clinical product candidate ATX-F8-117 has been granted Orphan Drug Status by the US Food and Drug Administration for the prevention or treatment of inhibitors in haemophilia A patients with inhibitors or at the risk of producing inhibitors. [More]
Global haemophilia therapeutics market estimated to reach $13.43 billion in 2019

Global haemophilia therapeutics market estimated to reach $13.43 billion in 2019

Increased uptake of recombinant therapies and prophylactic use, coupled with improved diagnosis of haemophilia, is expected to drive the associated therapeutics market. Recombinant therapies are likely to command a premium price and fuel growth in developed markets, while heightened access to treatment and larger patient volumes due to more funding for critical care are anticipated to contribute to demand in emerging markets. [More]

Added benefit of simoctocog alfa not proven for haemophilia A

Simoctocog alfa (trade name Nuwiq) has been approved since July 2014 for people with type A haemophilia, an inherited disorder that impairs blood clotting. The German Institute for Quality and Efficiency in Health Care examined in a dossier assessment whether this new drug offers an added benefit over the appropriate comparator therapy. Such an added benefit cannot be derived from the dossier, however, because the drug manufacturer did not submit any suitable data. [More]
Novo Nordisk reports positive data from Novoeight phase 3 study in people with haemophilia A

Novo Nordisk reports positive data from Novoeight phase 3 study in people with haemophilia A

Today, Novo Nordisk announced a new analysis of phase 3 data demonstrating people with haemophilia A who had the highest annualised bleeding rate (ABR) during initial treatment with Novoeight (Antihemophilic Factor [Recombinant]) showed the largest reduction in bleeding over the duration of treatment. [More]
Researchers set up innovative project to monitor physical activity of patients with haemophilia

Researchers set up innovative project to monitor physical activity of patients with haemophilia

The research group Intelligent Data Analysis Laboratory from the School of Engineering Universitat de València (UV) and a team of scientists at the Faculty of Physiotherapy led by Professor Felipe Querol have set up an innovative project for monitoring the physical activity of people with haemophilia through individual devices with the purpose of developing patterns which help to improve their quality of life and treatments. [More]
Apitope's ATX-F8-117 granted orphan medicinal product designation for treatment of haemophilia A

Apitope's ATX-F8-117 granted orphan medicinal product designation for treatment of haemophilia A

Apitope, the drug discovery and development company focused on disease-modifying treatments for patients with autoimmune and allergic diseases, announced today that the European Medicines Agency Committee for Orphan Medicinal Products has granted orphan medicinal product designation to ATX-F8-117 for the treatment of haemophilia A. [More]
EMA recommends marketing authorisation for Octapharma’s human cell line recombinant human FVIII, Nuwiq

EMA recommends marketing authorisation for Octapharma’s human cell line recombinant human FVIII, Nuwiq

Octapharma confirmed today the European Medicines Agency (EMA) has adopted a positive opinion towards human cell line recombinant human FVIII, Nuwiq®, recommending the granting of a marketing authorisation for the medicinal product for treatment and prophylaxis of bleeding (also during and after surgery) in paediatric and adult patients with haemophilia A (congenital FVIII deficiency). [More]
Study: Added benefit of turoctocog alfa is not proven

Study: Added benefit of turoctocog alfa is not proven

Turoctocog alfa (trade name: NovoEight) has been approved since November 2013 for the prevention and treatment of bleeding in patients with haemophilia A. [More]
Biogen Idec's ALPROLIX receives FDA approval for hemophilia B treatment

Biogen Idec's ALPROLIX receives FDA approval for hemophilia B treatment

Today Biogen Idec announced that the U.S. Food and Drug Administration has approved ALPROLIX [Coagulation Factor IX (Recombinant), Fc Fusion Protein], the first recombinant, DNA derived hemophilia B therapy with prolonged circulation in the body. [More]
Biogen Idec's ALPROLIX gets Health Canada approval for hemophilia B

Biogen Idec's ALPROLIX gets Health Canada approval for hemophilia B

Today Biogen Idec announced that Health Canada has approved ALPROLIX [Coagulation Factor IX (Recombinant), Fc Fusion Protein], for the control and prevention of bleeding episodes and routine prophylaxis in adults, and children aged 12 and older, with hemophilia B. [More]
OPKO Health consolidated revenues increase about 30% to $20.7 million in Q4 2013

OPKO Health consolidated revenues increase about 30% to $20.7 million in Q4 2013

OPKO Health, Inc., a multi-national biopharmaceutical and diagnostics company, today reported operating and financial results for its 2013 fourth quarter and full year ended December 31, 2013. [More]
Novo Nordisk to use Trialbee's services for e-recruitment of patients for clinical studies

Novo Nordisk to use Trialbee's services for e-recruitment of patients for clinical studies

Trialbee has developed a web based platform and methodology which enable the Novo Nordisk to perform fast and effective patient recruitment to specific clinical trials. Trialbee helps reduce the time required for recruitment resulting in important efficiency gains for the pharmaceutical company. [More]
FDA approves Baxter's FEIBA for prophylactic treatment of hemophilia patients with inhibitors

FDA approves Baxter's FEIBA for prophylactic treatment of hemophilia patients with inhibitors

Baxter International Inc. today announced that the United States Food and Drug Administration granted approval of Baxter's FEIBA [Anti-Inhibitor Coagulant Complex], the first and only FDA-approved treatment for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A or B who have developed inhibitors. [More]
Alnylam presents new pre-clinical data with ALN-AT3 for treatment of hemophilia and RBD

Alnylam presents new pre-clinical data with ALN-AT3 for treatment of hemophilia and RBD

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD), at the 55th Annual Meeting of the American Society of Hematology (ASH) held December 7 - 10, 2013 in New Orleans. [More]
Biogen, Sobi present new data supporting clinical, safety profile of ELOCTATE for hemophilia A

Biogen, Sobi present new data supporting clinical, safety profile of ELOCTATE for hemophilia A

Biogen Idec nd Swedish Orphan Biovitrum AB (publ) presented new data that support the clinical and safety profile of their long-lasting recombinant factor VIII candidate ELOCTATE for hemophilia A. [More]
Biogen, Sobi present new findings from Phase 3 trial of ALPROLIX at XXIV ISTH Congress

Biogen, Sobi present new findings from Phase 3 trial of ALPROLIX at XXIV ISTH Congress

Biogen Idec and Swedish Orphan Biovitrum AB (publ) today presented new findings for their long-lasting recombinant factor IX candidate ALPROLIX for hemophilia B at the XXIV International Society on Thrombosis and Haemostasis Congress in Amsterdam, The Netherlands. [More]
Alnylam Pharmaceuticals presents new pre-clinical data of ALN-AT3 at ISTH meeting

Alnylam Pharmaceuticals presents new pre-clinical data of ALN-AT3 at ISTH meeting

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data from its RNAi therapeutic program for the treatment of hemophilia and rare bleeding disorders. [More]
Santarus, Pharming Group announce FDA acceptance of RUCONEST BLA

Santarus, Pharming Group announce FDA acceptance of RUCONEST BLA

Santarus, Inc. and Pharming Group NV today announced that the U.S. Food and Drug Administration has accepted for filing the Biologics License Application for the investigational drug RUCONEST (recombinant human C1 esterase inhibitor) 50 IU/kg. [More]
Santarus, Pharming Group submit Biologics License Application to FDA for RUCONEST

Santarus, Pharming Group submit Biologics License Application to FDA for RUCONEST

Santarus, Inc. and Pharming Group NV today announced the submission of a Biologics License Application to the U.S. Food and Drug Administration to obtain marketing approval for RUCONEST (recombinant human C1 esterase inhibitor) 50 U/kg, an investigational drug for the treatment of acute angioedema attacks in patients with hereditary angioedema. [More]
Study sheds light on stem cell technique for treatment of common bleeding disorder

Study sheds light on stem cell technique for treatment of common bleeding disorder

Scientists have shed light on a common bleeding disorder by growing and analysing stem cells from patients' blood to discover the cause of the disease in individual patients. [More]
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