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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.
Coagadex now available for rare bleeding disorder patients in the UK

Coagadex now available for rare bleeding disorder patients in the UK

Bio Products Laboratory, Limited (BPL) today announced that Coagadex is now available for patients in the UK. [More]
European Medicines Agency approves first-ever treatment for hereditary factor X deficiency

European Medicines Agency approves first-ever treatment for hereditary factor X deficiency

Bio Products Laboratory, Limited (BPL) today announced that the European Medicines Agency has granted marketing authorisation for Coagadex. [More]
New €5.6 million project aims to develop cell-based haemophilia A therapy

New €5.6 million project aims to develop cell-based haemophilia A therapy

Loughborough University is working with a team of international partners on a new €5.6 million project to develop a cell based haemophilia A therapy. [More]
Improved gene therapy treatment shows promise in mice with cystic fibrosis

Improved gene therapy treatment shows promise in mice with cystic fibrosis

An improved gene therapy treatment can cure mice with cystic fibrosis (CF). Cell cultures from CF patients, too, respond well to the treatment. Those are the encouraging results of a study presented by the Laboratory for Molecular Virology and Gene Therapy at KU Leuven, Belgium. [More]
Simple, low risk blood test can detect foetal blood group and genetic conditions in unborn babies

Simple, low risk blood test can detect foetal blood group and genetic conditions in unborn babies

Research into a simple, accurate and low risk blood test that can detect foetal blood group, sex, and genetic conditions in unborn babies has been published in the international scientific journal, Clinical Chemistry. [More]
Approval to start trial of Videregen's replacement trachea technology

Approval to start trial of Videregen's replacement trachea technology

The INSPIRE consortium, led by Videregen Ltd, has received approval from the MHRA1 to start a UK clinical trial with its tissue engineered replacement trachea. [More]
FDA grants 12-year exclusivity to RUCONEST (C1 esterase inhibitor [recombinant])

FDA grants 12-year exclusivity to RUCONEST (C1 esterase inhibitor [recombinant])

Salix Pharmaceuticals and Pharming Group NV announced today that the U.S. Food and Drug Administration has granted 12 years of exclusivity to RUCONEST (C1 esterase inhibitor [recombinant]) 50 IU/kg. [More]
New report on different treatment strategies for haemophilia published

New report on different treatment strategies for haemophilia published

Numerous long-term randomized controlled trials (RCTs) have been conducted to investigate the long-term, factor concentrate-based treatment of patients with severe haemophilia A or B, despite the rareness of the diseases and the lack of incentives for pharmaceutical companies. [More]
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]
Baxter submits BAX 855 NDA to Japan's MHLW for treatment of hemophilia A patients

Baxter submits BAX 855 NDA to Japan's MHLW for treatment of hemophilia A patients

Nektar Therapeutics reported today that partner Baxter International Inc. announced that the company has submitted a new drug application (NDA) to Japan's Ministry of Health, Labour and Welfare for the approval of BAX 855, an investigational, extended half-life recombinant factor VIII (rFVIII) treatment based on ADVATE [Antihemophilic Factor (Recombinant)] for patients over 12 years of age with hemophilia A. [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]
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