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

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. [More]
European collaboration to design a biomimetic bioartificial liver

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. [More]
Study describes new method that synthesizes novel anti-HIV protein

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. [More]
EvaluatePharma data shows poor R&D productivity measures both in quality, quantity

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. [More]
Grifols receives FDA approval for Alphanate to treat vCJD

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. [More]
Inspiration presents IB1001 PK data for treatment of hemophilia B

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. [More]
Inspiration presents IB1001 PK data at European haemophilia congress

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. [More]
LFB receives positive review feedback for closing of Willfact 1000 IU Mutual Recognition Procedure

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. [More]
Octapharma initiates Human-cl rhFVIII clinical study for severe hemophilia A

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. [More]
Octapharma sponsors symposium on inhibitors in Haemophilia A

Octapharma sponsors symposium on inhibitors in Haemophilia A

In a showing of its continued commitment to eradicating today's major complication in the treatment of haemophilia with FVIII concentrates − i.e. anti-FVIII neutralising antibodies − Octapharma AG recently sponsored a symposium, "Inhibitors in Haemophilia A: Prevention, Current Management and Personalised Therapy Perspectives", on July 13, 2010 at the World Federation of Haemophilia's (WFH's) yearly congress. [More]
NKTreg white blood cells migrate into liver, suppress immune responses: Research

NKTreg white blood cells migrate into liver, suppress immune responses: Research

After a transplant - and to assure that the new organ is not rejected - patients are put on life-long therapy to suppress their immune system, which, nevertheless, needs to be left intact enough to be able to defend the body against all kinds of disease. A tricky balance as the many rejected organs attest. But a discovery by Maria Monteiro and Luis Graça, two Portuguese scientists, could change all this, at least for the liver. [More]
Pfizer to present results of hemophilia studies at WFH 2010 Congress

Pfizer to present results of hemophilia studies at WFH 2010 Congress

Pfizer Inc, the world's leading biopharmaceutical company, today announced that the results of a number of hemophilia studies will be presented at the World Federation of Hemophilia 2010 Congress taking place July 10-14, 2010, in Buenos Aires, Argentina. Key research includes a pre-clinical evaluation of recombinant factor Xa as a potential new approach to restoring hemostasis, as well as a study assessing the potential for an engineered recombinant factor VIIa molecule to improve therapeutic outcomes in mouse models of hemophilia. [More]