Factor X Deficiency News and Research

RSS

Opportunities and risks of direct-to-consumer genetic health risk tests

Accurate genetic testing stands to transform modern medicine by offering effective, personalized treatment.

13 Apr 2017

FDA authorizes marketing of first direct-to-consumer GHR tests for certain diseases

The U.S. Food and Drug Administration today allowed marketing of 23andMe Personal Genome Service Genetic Health Risk (GHR) tests for 10 diseases or conditions.

6 Apr 2017

Penn researchers use CRISPR/Cas9 gene targeting approach to treat hemophilia B in mice

CRISPR/Cas9, a powerful genome editing tool, is showing promise for efficient correction of disease-causing mutations.

1 Dec 2016

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.

7 Jun 2016

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.

31 Mar 2016

Idelvion approved for use in children and adults with Hemophilia B

The U.S. Food and Drug Administration today approved Idelvion, Coagulation Factor IX (Recombinant), Albumin Fusion Protein, for use in children and adults with Hemophilia B. Idelvion is the first coagulation factor-albumin fusion protein product to be approved, and the second Factor IX fusion protein product approved in the U.S. that is modified to last longer in the blood.

7 Mar 2016

Gene therapy holds considerable potential for safe, effective treatment of people with factor VII deficiency

Hematology researchers have used a single injection of gene therapy to correct a rare bleeding disorder, factor VII deficiency, in dogs. This success in large animals holds considerable potential for a safe, effective and long-lasting new treatment in humans with the same bleeding disorder.

21 Jan 2016

Novel gene therapy treatment proves safe, effective for factor VII deficiency

A single injection. That's all someone with a factor VII deficiency would need for a life-long cure, thanks to a new gene therapy treatment developed in a collaboration of researchers at the University of North Carolina and The Children's Hospital of Philadelphia.

20 Jan 2016

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

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 rIX-FP granted Orphan Drug Designation to treat hemophilia B

CSL Behring announced today that the company has been granted Orphan Drug Designation by the United States Food and Drug Administration for its novel recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP).

8 Jun 2012

Chinese sFDA approves Baxter's ADVATE to treat hemophilia A

Baxter International Inc. today announced the approval of ADVATE [Recombinant Human Coagulation Factor VIII for injection] for the control and prophylaxis of bleeding episodes in individuals with hemophilia A (congenital factor VIII deficiency) in China by the State Food and Drug Administration.

From Baxter International Inc. 17 May 2012

U.S. FDA approves Corifact to prevent bleeding in people with Factor XIII deficiency

The U.S. Food and Drug Administration today approved Corifact, the first product intended to prevent bleeding in people with the rare genetic defect congenital Factor XIII deficiency.

18 Feb 2011

Researchers investigate Human-cl rhFVIII factor for severe hemophilia A treatment

Octapharma AG, one of the largest human protein products manufacturers in the world, and Octapharma USA today announced that patients diagnosed with severe hemophilia A have started treatment in a multi-center study that is researching the use of the first recombinant Factor VIII (rFVIII) derived from a human cell line (Human-cl rhFVIII).

2 Nov 2010

FDA approves Pfizer's Prefilled Dual-Chamber Syringe for XYNTHA administration in hemophilia A patients

Pfizer Inc. announced today that the U.S. Food and Drug Administration has granted approval of the use of a Prefilled Dual-Chamber Syringe for administration of XYNTHA® Antihemophilic Factor (Recombinant) Plasma/Albumin-Free to hemophilia A patients. XYNTHA is a recombinant factor VIII product indicated for both the control and prevention of bleeding episodes in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia) and for surgical prophylaxis in patients with hemophilia A.

9 Aug 2010

Alexion's Soliris may benefit patients with thrombotic microangiopathy from uncontrolled complement activation

Researchers reported today that Soliris® (eculizumab), a first-in-class terminal complement inhibitor developed by Alexion Pharmaceuticals, Inc., may provide clinical benefits to patients with thrombotic microangiopathy (TMA) resulting from uncontrolled complement activation. TMA, the formation of blood clots in capillaries and small arteries, can lead to life-threatening damage in multiple organs including kidney failure, thrombocytopenia (abnormally low platelet count) and anemia.

15 Jun 2010

FDA approves new formulation of genetically engineered version of Factor VIIa plasma protein

The U.S. Food and Drug Administration today approved a new formulation of the genetically engineered version of Factor VIIa, a plasma protein essential for the clotting of blood. The new formulation allows the product to be stored at room temperature (up to 81 degrees Fahrenheit) for up to two years.

9 May 2008

XYNTHA approved for teatment of hemophilia A by FDA

Wyeth Pharmaceuticals, a division of Wyeth ( NYSE:WYE) , announced today that it has received approval from the U.S. Food and Drug Administration for XYNTHA(TM) (Antihemophilic Factor [Recombinant], Plasma/Albumin-Free), a recombinant factor VIII product, for patients with hemophilia A for both the control and prevention of bleeding episodes and surgical prophylaxis.

22 Feb 2008

Prophylaxis effective at preventing joint bleeds and preserving joint function and structure in young boys with hemophilia A

According to a new landmark study published in The New England Journal of Medicine, infusions of clotting factor VIII used preventively can significantly reduce the risk of developing joint damage associated with joint bleeding in young children with hemophilia A.

9 Aug 2007