Bleeding Disorder News and Research

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FDA approves recombinant von Willebrand factor for treating bleeding episodes in adults with VWD

The U.S. Food and Drug Administration today approved Vonvendi, von Willebrand factor (Recombinant), for use in adults 18 years of age and older who have von Willebrand disease (VWD). Vonvendi is the first FDA-approved recombinant von Willebrand factor, and is approved for the on-demand (as needed) treatment and control of bleeding episodes in adults diagnosed with VWD.

8 Dec 2015

Rice scientists solve long-standing mystery about hemophilia protein

Rice University scientists have solved a long-standing mystery about where the body stores and deploys blood-clotting factor VIII, a protein that about 80 percent of hemophiliacs cannot produce due to genetic defects.

3 Nov 2015

Coagadex now approved by FDA for patients with hereditary Factor X deficiency

The U.S. Food and Drug Administration today approved Coagadex, Coagulation Factor X (Human), for hereditary Factor X (10) deficiency. Until today's orphan drug approval, no specific coagulation factor replacement therapy was available for patients with hereditary Factor X deficiency.

21 Oct 2015

First patient enrolled in CSL Behring's rVIIa-FP Phase II/III study to treat patients with hemophilia A or B with inhibitors

CSL Behring announced today that the first patient has been enrolled in its Phase II/III clinical study evaluating the pharmacokinetics (PK), efficacy, and safety of the company's recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP) for on-demand treatment in patients with congenital hemophilia A or B who have developed an inhibitor to factor VIII or factor IX replacement therapy.

26 Aug 2015

Alnylam presents positive Phase 1 ALN-AT3 trial results in hemophilia at ISTH 2015 Congress

Additional study results from 12 hemophilia A and B subjects demonstrated that subcutaneous administration of ALN-AT3 achieved potent and dose-dependent knockdown of AT of up to 86%. AT knockdown was highly durable, with effects lasting over two months after the last dose, supporting further evaluation of a once-monthly subcutaneous dose regimen.

24 Jun 2015

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.

9 Jun 2015

CSL Behring awarded NHF's 2015 Corporate Leadership Award

The National Hemophilia Foundation has awarded CSL Behring its 2015 Corporate Leadership Award as recognition for the company's longstanding and unwavering commitment to advancing science and improving the care of the bleeding disorders community. The award was accepted by Paul Perreault, CEO and Managing Director, CSL Limited, during the NHF Annual Spring Soiree in New York City on May 21.

24 May 2015

CMC Biologics to supply bulk drug substance for Emergent BioSolutions' IXINITY

CMC Biologics, Inc., a global CMO dedicated to process development and production of biopharmaceuticals for clinical and commercial use, today announced that it will supply bulk drug substance for Emergent BioSolutions' recently FDA-approved product, IXINITY® [coagulation factor IX (recombinant)].

5 May 2015

Scientists identify missing genetic link in common variable immunodeficiency disorder

In the largest genetic study to date of a challenging immunodeficiency disorder, scientists have identified a gene that may be a "missing link" between overactive and underactive immune activity. The gene candidate also plays a key role in autoimmune diseases such as type 1 diabetes, rheumatoid arthritis and allergies.

23 Apr 2015

'Turbocharged' protein holds promise in hemophilia gene therapy

Using gene therapy to produce a mutant human protein with unusually high blood-clotting power, scientists have successfully treated dogs with the bleeding disorder hemophilia, without triggering an unwanted immune response.

13 Mar 2015

Study: New gene therapy safe, effective for patients with hemophilia B

A multi-year, ongoing study suggests that a new kind of gene therapy for hemophilia B could be safe and effective for human patients. Published in the journal Science Translational Medicine, the research showed that a reprogrammed retrovirus could successfully transfer new factor IX (clotting) genes into animals with hemophilia B to dramatically decrease spontaneous bleeding.

12 Mar 2015

Bayer announces FDA acceptance of BAY 81-8973 BLA for treatment of hemophilia A

Bayer HealthCare today announced that the U.S. Food and Drug Administration has accepted the company's Biologics License Application for BAY 81-8973, a recombinant Factor VIII compound. Bayer is seeking FDA approval of the investigational compound, proposed trade name Kovaltry, for the treatment of hemophilia A in children and adults.

4 Mar 2015

CSL Behring donates protein therapies to World Federation of Hemophilia

In recognition of Rare Disease Day and as part of its ongoing commitment to the global bleeding disorders community, CSL Behring announced today that it is donating 2 million international units (IUs) of protein therapies to the World Federation of Hemophilia.

28 Feb 2015

Spark Therapeutics, Pfizer partner to develop SPK-FIX for potential treatment of hemophilia B

Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating genetic diseases, announced today that it has entered into a global collaboration with Pfizer Inc. for the development and potential commercialization of SPK-FIX, a development program advancing proprietary, bio-engineered adeno-associated virus (AAV) vectors for the potential treatment of hemophilia B.

8 Dec 2014

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.

From Novo Nordisk 7 Dec 2014

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.

25 Nov 2014

Gene therapy transforms life for men with severe form of hemophilia B

Gene therapy developed at St. Jude Children's Research Hospital, University College London and the Royal Free Hospital has transformed life for men with a severe form of hemophilia B by providing a safe, reliable source of the blood clotting protein Factor IX that has allowed some to adopt a more active lifestyle, researchers reported.

19 Nov 2014

Cases of Ebola virus in West Africa continue to rise

Since the Ebola virus outbreak was first reported in March there have been nearly 2,000 cases and over 1,000 deaths in Guinea, Liberia, Nigeria, and Sierra Leone. This makes it the most serious Ebola epidemic to date.

15 Aug 2014

Bayer, Dimension Therapeutics partner to develop new gene therapy for hemophilia A

Dimension Therapeutics, a company focused on developing novel adeno-associated virus (AAV) gene therapy treatments for rare diseases, today announced it has entered into a collaboration with Bayer HealthCare (Bayer) for the development and commercialization of a novel gene therapy for the treatment of hemophilia A.

23 Jun 2014

FDA approves Bayer's Kogenate FS antihemophilic factor VIII to prevent bleeding episodes in adults with hemophilia A

Bayer HealthCare announced today that the U.S. Food and Drug Administration (FDA) has approved a new indication for Bayer's Kogenate® FS antihemophilic factor VIII (recombinant), for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults with hemophilia A.

12 May 2014