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THE CONSERVATORY OF MEDICAL ARTS AND SCIENCES
XVII
THE SEARCHLIGHT MESSENGER
THE SEARCHLIGHT MESSENGER
Blog
Von Willebrand Disease Phase 3 Trial Reports 100% Bleed Control
| Posted on August 12, 2015 at 11:02 AM |
 Von Willebrand disease (VWD) is an inherited, genetically and
clinically heterogeneous hemorrhagic disorder caused by a deficiency or
dysfunction of the protein called Von Willebrand factor (VWF). This protein is a necessary step in the coagulation cascade, but also involved in the initiation of platelet aggregation for proper blood clotting. Consequently, defective VWF interaction between platelets and the vessel
wall impairs primary hemostasis. Von Willibrand factor 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
vascular bed endothelial cells. VWF performs two major roles in hemostasis, wether intrinsic or extrinsic. First, it
mediates the adhesion of platelets to sites of vascular injury. Second,
it binds and stabilizes the procoagulant protein factor VIII (FVIII). The disease is divided into three major categories: Partial Quantitative Deficiency (type I), Qualitative Deficiency (type II), and Total Deficiency (type III). VWD type II is further divided into
four variant conditions (IIA, IIB, IIN, IIM), based on characteristics of
dysfunctional VWF. According
to data pre-published online August 3rd, 2015, in Blood, successful management
of bleeding episodes were observed in 100% of subjects treated with BAX 111 for
von Willebrand disease (VWD) during a recently completed Phase 3 clinical
trial. Bleeding-episode-management success was the primary endpoint
of the clinical trial. BAX 111 is a highly purified recombinant von
Willebrand factor (VWF) analog manufactured by Baxalta, Inc.
VWD is a rare, inherited, incurable, gene-based bleeding disorder in which a
missing or defective clotting protein (VWF) fails to bind with platelets in
blood vessel walls. Normally, a blood-vessel tear initiates
bleeding and VWF assists in the repair. When VWF is absent or under-represented, the formation of platelet plugs are
inhibited during the clotting process, resulting in excessive bleeding and easy
bruising. In more severe forms of VWD, the bleeding can be
life-threatening and require emergency treatment. Efficacy and Safety of BAX 111 Gill et al derived their findings
from a Phase 3, multicenter, international, open-label study which evaluated
the safety, efficacy and pharmacokinetics of BAX 111 in 37 patients with severe
VWD.
Study participants evidenced a mean efficacy rating of < 2.5 on a 4-point
scale wherein lower numbers correlated with a higher degree of bleed control
(see sidebar below). Bleed control for all treated bleeding events (N=192
bleeds in 22 subjects) was rated as good or excellent (96.9% excellent; 119/122
minor, 59/61 moderate, and 6/7 major bleeds). In 81.8% of bleeds, 1 infusion
was sufficient to attain control. For major bleeds, the infusion median was Sidebar: Hemostatic Efficiency Rating Scale 1 (Excellent) • Minor and Moderate Bleeding Events o
Actual number of infusion less than or equal to estimated number of infusion
required to treat that bleeding episode. No additional
VWF-containing/coagulation factor containing product required. • Major
Bleeding Events o
Actual number of infusion less than or equal to estimated number of infusion
required to treat that bleeding episode. No additional
VWF-containing/coagulation factor containing product required. 2 (Good) • Minor and Moderate Bleeding Events o 1 to
2 infusions greater than estimated required to control that bleeding episode.
No additional VWF-containing/coagulation factor containing product required. • Major
Bleeding Events o Less
than 1.5x greater than estimated required to control that bleeding episode.
No additional VWF-containing/coagulation factor containing product required. 3 (Moderate) • Minor and Moderate Bleeding Events o 3 or
more infusions greater than estimated used to control that bleeding event. No
additional VWF-containing/coagulation factor containing product required. • Major
Bleeding Events o
Greater than or equal to 1.5x greater than estimated used to control that
bleeding event. No additional VWF-containing/coagulation factor containing
product required. 4 (None) • Minor and Moderate Bleeding Events o
Severe uncontrolled bleeding or intensity of bleeding not changed. Additional
VWF-containing/coagulation factor containing product required. • Major
Bleeding Events o
Severe uncontrolled bleeding or intensity of bleeding not changed. Additional
VWF-containing/coagulation factor containing product required.References: Safety and tolerability outcomes - evaluated via clinical assessments of
adverse events, hematology panels, coagulation panels, serum chemistry,
urinalysis, viral serology and immunological assessments - were also
encouraging. With the exception of 1 patient, adverse events were minor or
unrelated to treatment. No thrombotic events or severe allergic reactions
occurred, and none of the participants developed anti-VWF binding or
neutralizing antibodies to VWF.
Researchers concluded that the data offer evidence that BAX 111 is “safe and
hemostatically effective in severe VWD patients in a variety of clinical
bleeding presentations.” Addressing a Pressing Therapeutic
Need “Von
Willebrand disease is the most common hereditary bleeding disorder, yet few
treatment options exist,” noted John Orloff, MD, Head of Research &
Development and Chief Scientific Officer, Baxalta. BAX 111, Dr. Orloff
asserted, “has the potential to transform the standard of care for patients
with severe von Willebrand disease by offering an effective, individualized
treatment option.”
Both the FDA and the European Medicines Agency granted orphan drug designation
to BAX 111 back in November 2010.2 Currently, BAX 111 remains under FDA review,
a pending Biologics License Application having been filed in December 2014.5
While no official Prescription Drug User Fee Act (PDUFA) date has been set,
December 22, 2015, has been cited by industry insiders as a speculative
estimation. The PDUFA date is an FDA approval deadline for new
drugs. If approved, BAX 111 would become the first recombinant
replacement treatment indicated for the management of VWD-related bleeding
episodes. References
 |
Categories: Clinical Update
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