Haemophilia A News and Research

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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.

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.

10 Dec 2013

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.

4 Jul 2013

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.

3 Jul 2013

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.

2 Jul 2013

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.

18 Jun 2013

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.

17 Apr 2013

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.

6 Apr 2013

Biogen Idec, Sobi release data from rFVIIIFc and rFIXFc phase 3 trials on hemophilia

Biogen Idec and Swedish Orphan Biovitrum (Sobi) released data that confirmed the ability of investigational recombinant factors VIII Fc fusion protein (rFVIIIFc) and IX Fc fusion protein (rFIXFc) to provide long-lasting protection from bleeding with fewer injections than are required with the current standard of care for people with hemophilia.

10 Feb 2013

Researchers to develop new diagnostic tools and treatments for people with rare diseases

A multi-million Euro initiative is bringing together researchers from across the world to develop new diagnostic tools and new treatments for people with rare diseases and to connect research data in this area on a global scale.

25 Jan 2013

Interim results from Inspiration’s OBI-1 and IB1001 hemophilia clinical study

Inspiration Biopharmaceuticals, Inc. (Inspiration) announced interim clinical results for the company's two lead product candidates, an intravenous recombinant porcine FVIII (OBI-1) in the treatment of serious bleeds in acquired hemophilia A (AHA) and an intravenous recombinant FIX (IB1001) for the treatment and prevention of bleeding in children less than 12 years of age with hemophilia B.

13 Dec 2012

Positive results from Santarus and Pharming’s RUCONEST Phase III trial on HAE

Santarus, Inc. and Pharming Group NV today announced that their pivotal Phase III clinical study to evaluate the safety and efficacy of the investigational drug RUCONEST (recombinant human C1 esterase inhibitor) 50 U/kg for the treatment of acute attacks of angioedema in patients with Hereditary Angioedema (HAE) met the primary endpoint of time to beginning of symptom relief.

7 Nov 2012

Hemostatic testing may explain heavy menstrual bleeding

Results of a US study show that hemostatic screening tests are important in the evaluation of young women with heavy menstrual bleeding.

24 Oct 2012

European collaboration to design a biomimetic bioartificial liver

Medicyte, The Electrospinning Company and the Universities of Manchester and Pisa launch a European wide unique project with the aim to design a biomimetic bioartificial liver (Re-Liver). The company GABO:mi ensures professional project management.

2 Jul 2012

Study describes new method that synthesizes novel anti-HIV protein

The most abundant and important molecules in all living organisms are proteins; after all they manage to participate in every single one of life's essential reactions. So it is easy to see why scientists have been making such a fuss trying to learn how to synthesise them in laboratory as this would provide them with a tool of extraordinary potential. Unfortunately, this has not proved easy.

13 Dec 2011

EvaluatePharma data shows poor R&D productivity measures both in quality, quantity

Pharmaceutical R&D productivity is consistently undercounted. Most reports fail to fully count the number of new drugs that reach the market, missing out on billions of dollars of sales generated by R&D investment, an analysis of EvaluatePharma data shows.

14 Mar 2011

Grifols receives FDA approval for Alphanate to treat vCJD

Grifols, a global healthcare company and biopharmaceutical manufacturer based in Barcelona, Spain today announced that it received approval from the US Food and Drug Administration for revised labeling for Alphanate Antihemophilic Factor/von Willebrand Factor Complex indicating that certain manufacturing steps have been shown to reduce the infectivity of a experimental TSE agent that is a model for variant Creutzfeldt Jakob Disease.

5 Mar 2011

Inspiration presents IB1001 PK data for treatment of hemophilia B

Inspiration Biopharmaceuticals, today presented pharmacokinetic data on its lead product, IB1001, a recombinant factor IX for the treatment and prevention of bleeding in individuals with hemophilia B.

3 Feb 2011

Inspiration presents IB1001 PK data at European haemophilia congress

Ipsen today announced that its partner Inspiration Biopharmaceuticals, Inc. (Inspiration) presented pharmacokinetic (PK) data on its lead product, IB1001, a recombinant factor IX (FIX) for the treatment and prevention of bleeding in individuals with hemophilia B.

3 Feb 2011

LFB receives positive review feedback for closing of Willfact 1000 IU Mutual Recognition Procedure

LFB received from the German regulatory authority (Paul-Ehrlich-Institute) a positive review feedback for the closing of the Mutual Recognition Procedure (MRP) of Willfact® 1000 IU with Germany as the reference member state.

22 Dec 2010

Octapharma initiates Human-cl rhFVIII clinical study for severe hemophilia A

Octapharma, one of the largest human protein products manufacturers in the world, today announced that patients diagnosed with severe hemophilia A have started treatment with the first recombinant Factor VIII derived from a human cell line (Human-cl rhFVIII). Researchers are investigating pharmacokinetics, efficacy, safety and immunogenicity of Human-cl rhFVIII for previously treated patients with severe hemophilia A.

28 Oct 2010