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Posts Tagged ‘heritable diseases’

von Willebrand Factor

Larry H. Bernstein, MD, FCAP, Curator

LPBI

 

FDA approves first recombinant von Willebrand factor to treat bleeding episodes

Dr. Anthony Melvin Castro

 

 

12/08/2015 02:44
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.
Company Baxalta Inc.
Description Recombinant human von Willebrand factor (vWF)
Molecular Target von Willebrand factor (vWF)
Mechanism of Action
Therapeutic Modality Biologic: Protein
Latest Stage of Development Registration
Standard Indication Bleeding
Indication Details Treat and prevent bleeding episodes in von Willebrand disease (vWD) patients; Treat von Willebrand disease (vWD)
Regulatory Designation U.S. – Orphan Drug (Treat and prevent bleeding episodes in von Willebrand disease (vWD) patients);
EU – Orphan Drug (Treat and prevent bleeding episodes in von Willebrand disease (vWD) patients);
Japan – Orphan Drug (Treat and prevent bleeding episodes in von Willebrand disease (vWD) patients)

 

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.

VWD is the most common inherited bleeding disorder, affecting approximately 1 percent of the U.S. population. Men and women are equally affected by VWD, which is caused by a deficiency or defect in von Willebrand factor, a protein that is critical for normal blood clotting. Patients with VWD can develop severe bleeding from the nose, gums, and intestines, as well as into muscles and joints. Women with VWD may have heavy menstrual periods lasting longer than average and may experience excessive bleeding after childbirth.

“Patients with heritable bleeding disorders should meet with their health care provider to discuss appropriate measures to reduce blood loss,” said Karen Midthun, M.D., director of the FDA’s Center for Biologics Evaluation and Research. “The approval of Vonvendi provides an additional therapeutic option for the treatment of bleeding episodes in patients with von Willebrand disease.”

The safety and efficacy of Vonvendi were evaluated in two clinical trials of 69 adult participants with VWD. These trials demonstrated that Vonvendi was safe and effective for the on-demand treatment and control of bleeding episodes from a variety of different sites in the body. No safety concerns were identified in the trials. The most common adverse reaction observed was generalized pruritus (itching).

The FDA granted Vonvendi orphan product designation for these uses.Orphan product designation is given to drugs intended to treat rare diseases in order to promote their development.

Vonvendi is manufactured by Baxalta U.S., Inc., based in Westlake Village, California.

 

von Willebrand Disease

Author: Eleanor S Pollak; Chief Editor: Srikanth Nagalla

Von Willebrand disease (vWD) is a common, inherited, genetically and clinically heterogeneous hemorrhagic disorder caused by a deficiency or dysfunction of the protein termed von Willebrand factor (vWF). Consequently, defective vWF interaction between platelets and the vessel wall impairs primary hemostasis.

vWF, a large, multimeric glycoprotein, circulates in blood plasma at concentrations of approximately 10 mg/mL. In response to numerous stimuli, vWF is released from storage granules in platelets and endothelial cells. It performs two major roles in hemostasis. First, it mediates the adhesion of platelets to sites of vascular injury. Second, it binds and stabilizes the procoagulant protein factor VIII (FVIII). (See Etiology.)

vWD is divided into three major categories: (1) partial quantitative deficiency (type I), (2) qualitative deficiency (type II), and (3) total deficiency (type III). vWD type II is further divided into four variants (IIA, IIB, IIN, IIM), based on characteristics of dysfunctional vWF. These categories correspond to distinct molecular mechanisms, with corresponding clinical features and therapeutic recommendations.

For discussion of vWD in children, see Pediatric Von Willebrand Disease.

http://emedicine.medscape.com/article/206996-overview

 

 

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