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.

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

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.

13 Sep 2010

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.

11 Aug 2010

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.

9 Jul 2010

Bayer HealthCare to present BAY86-6150 rFVIIa Phase I clinical data at XXIX WFH International Congress

Phase I clinical data for BAY86-6150, a novel recombinant factor VIIa protein (rFVIIa) being developed by Bayer HealthCare, will be presented at the XXIX International Congress of the World Federation of Hemophilia (WFH) to be held in Buenos Aires, Argentina from Saturday, July 10-Wednesday, July 14.

9 Jul 2010

Simple blood test may help detect chromosomal abnormalities in developing foetus

Researchers in The Netherlands believe they are on the verge of developing a simple, prenatal blood test that would be able to detect accurately chromosomal abnormalities in the developing foetus.

29 Jun 2010

Non-Invasive prenatal blood test to detect chromosomal abnormalities in foetus

Researchers in The Netherlands believe they are on the verge of developing a simple, prenatal blood test that would be able to detect accurately chromosomal abnormalities in the developing foetus. At present, the only reliable way to do this is through amniocentesis or chorionic villus sampling, both of which are invasive and carry the risk of triggering a miscarriage.

29 Jun 2010

Ban on Gay men from donating blood to be reviewed in light of new evidence

Australian Red Cross Blood Service (ARCBS) has recently announced plans for a review of Australia's ban on sexually-active gay men donating blood. Canada, Scotland, Sweden and New Zealand have already conducted such reviews and the US is soon to follow suit.

14 Apr 2010

Study may pave for effective treatments for blood clotting disorders

A key protein that causes the blood to clot is produced by blood vessels in the lungs and not just the liver, according to new research published today in the journal PLoS One, led by scientists at Imperial College London.

11 Feb 2010

Lysteda tablets to treat menorrhagia approved by the FDA

The U.S. Food and Drug Administration today approved Lysteda tablets (tranexamic acid), the first non-hormonal product cleared to treat heavy menstrual bleeding (menorrhagia). Lysteda works by stabilizing a protein that helps blood to clot.

24 Nov 2009

Amsterdam Molecular Therapeutics provides third-quarter 2009 business updates

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today provides its non-audited business update in compliance with the EU transparency directive. This report summarizes material events and AMT's financial position for the third quarter of 2009.

18 Nov 2009

Lack of hemophilia treatment in the developing world costing lives

When modern medicine finds a way to treat a medical condition, people often think that the problem is solved. But we also have to find ways to get that treatment into the hands of those who need it. For example, new research from North Carolina State University shows that much more needs to be done to help get existing treatment to hemophilia patients in the developing world, and that the current lack of treatment there is costing lives.

16 Nov 2009

Amorfix Life Sciences reports operating results for the three months ended September 30, 2009

Amorfix Life Sciences, a company focused on misfolded protein diseases, today reported its second quarter operating results and provided an update on the Company's diagnostic and therapeutic programs.

13 Nov 2009

Gene therapy saves two children with adrenoleukodystrophy

The ELA association and Zinedine Zidane, its emblematic ambassador, are proud to announce a world premiere: the results regaring the gene therapy in adrenoleukodystrophy conducted in France have just been published in the prestigious journal Science. Two children have been treated and their diseases have been halted. The children are doing well, which is unexpected for a disease destroying the brain in a few months. This discovery opens up treatment perspectives for numerous widespread diseases.

6 Nov 2009

LFB Biotechnologies exercises its option to purchase $12.8 million of stocks in GTC Biotherapeutics

GTC Biotherapeutics, Inc. announced that LFB Biotechnologies, a wholly owned subsidiary of LFB S.A. (Laboratoire français du Fractionnement et des Biotechnologies S.A., Les Ulis, France) and a strategic shareholder in GTC, has exercised its option to purchase $12.8 million of additional convertible preferred stock under the terms described in the financing agreements approved by GTC shareholders in July 2009. This transaction provides $6.4 million of new cash proceeds to GTC.

2 Nov 2009