VWD

Octapharma to present clinical and scientific data that advances our understanding and treatment of bleeding disorders at the 17th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), which takes place in Frankfurt, Ge

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Wednesday, January 17, 2024

The study shows that wilate® prophylaxis is highly effective at reducing bleeding rates in children and adults with all types of VWD and across examined bleeding sites.

Key Points: 
  • The study shows that wilate® prophylaxis is highly effective at reducing bleeding rates in children and adults with all types of VWD and across examined bleeding sites.
  • The results provide compelling evidence for the use of regular prophylaxis in people with VWD.
  • "The new data from the WIL-31 study provide strong evidence for the use of wilate® prophylaxis in people with all types of VWD.
  • In such instances, indirect treatment comparisons can be used to compare the effects of different treatments.

Human medicines European public assessment report (EPAR): Veyvondi, vonicog alfa, Date of authorisation: 31/08/2018, Revision: 9, Status: Authorised

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Tuesday, January 2, 2024

Human medicines European public assessment report (EPAR): Veyvondi, vonicog alfa, Date of authorisation: 31/08/2018, Revision: 9, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Veyvondi, vonicog alfa, Date of authorisation: 31/08/2018, Revision: 9, Status: Authorised

Octapharma USA: FDA Grants Expanded Approval to wilate® as the First VWF Concentrate for Prophylaxis in All Types of VWD

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Tuesday, December 5, 2023

PARAMUS, N.J., Dec. 5, 2023 /PRNewswire/ -- Octapharma USA announced the expansion of the U.S. Food and Drug Administration (FDA) approval for wilate®, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection. The approved label now includes routine prophylaxis aimed at reducing the frequency of bleeding episodes in adults and children aged 6 and older with any type of von Willebrand disease (VWD), the most prevalent bleeding disorder in the United States. Wilate® is the first von Willebrand factor (VWF) concentrate indicated for prophylactic treatment across all forms of VWD, marking a significant milestone in the field.

Key Points: 
  • Wilate® is the first von Willebrand factor (VWF) concentrate indicated for prophylactic treatment across all forms of VWD, marking a significant milestone in the field.
  • "The approval of wilate® for VWD prophylaxis is a welcome new treatment option that can be life-saving for many patients.
  • Increased use of VWF prophylaxis in VWD patients may lead to improved patient care and a reduced burden of disease."
  • In tandem with this approval, Octapharma USA extends its commitment to patient care through the Factor My Way patient support program.

Pathway to Cures Announces Inaugural Fund Update

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Thursday, November 2, 2023

Pathway to Cures (P2C) held its inaugural fund meeting on Saturday, October 28th in Chicago, Illinois.

Key Points: 
  • Pathway to Cures (P2C) held its inaugural fund meeting on Saturday, October 28th in Chicago, Illinois.
  • Pathway to Cures, an affiliate of the National Bleeding Disorders Foundation (NBDF), is a venture philanthropy fund focused on investing in early-stage companies developing cures, therapies, or enabling technologies in support of the inheritable blood and bleeding disorders community.
  • “Transcutaneous auricular neurostimulation (tAN) is a non-invasive method demonstrating great promise for treating various diseases.
  • “We have reviewed 114 companies over the past few months, whose promising research will accelerate the treatments and cures for rare diseases in the years to come.

Pathway to Cures Announces Venture Investment in Five Liters

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Monday, October 16, 2023

Pathway to Cures (P2C) announced today its investment of $500,000 in Five Liters, a subsidiary of Spark Biomedical, to develop therapies for treating inheritable blood and bleeding disorders.

Key Points: 
  • Pathway to Cures (P2C) announced today its investment of $500,000 in Five Liters, a subsidiary of Spark Biomedical, to develop therapies for treating inheritable blood and bleeding disorders.
  • Pathway to Cures, an affiliate of the National Bleeding Disorders Foundation, is a venture philanthropy fund focused on early-stage companies developing cures, therapies, or enabling technologies in support of the inheritable blood and bleeding disorders community.
  • This significant milestone is made possible through Five Liters' collaboration with Northwell Health’s Feinstein Institutes for Medical Research in New York.
  • Dr. Recht is Chief Medical and Scientific Officer at both Pathway to Cures and the National Bleeding Disorders Foundation (NBDF) and chairs the Pathway to Cures Scientific Advisory Group.

Five Liters sets clinical sights on novel bioelectric treatment for von Willebrand Disease

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Thursday, July 6, 2023

Five Liters, a subsidiary of Spark Biomedical, is dedicated to exploring the hemostatic effects of vagus nerve stimulation (VNS), specifically transcutaneous auricular neurostimulation (tAN).

Key Points: 
  • Five Liters, a subsidiary of Spark Biomedical, is dedicated to exploring the hemostatic effects of vagus nerve stimulation (VNS), specifically transcutaneous auricular neurostimulation (tAN).
  • Dr. Navid Khodaparast, Chief Science Officer of Spark and Five Liters stated “Von Willebrand Disease is an inherited bleeding disorder in which blood does not clot properly, leading to bleeding events and joint problems.
  • At Five Liters, our primary clinical initiative is to demonstrate that tAN therapy can safely alleviate menorrhagia in females with VWD.
  • Thanks to this partnership and the Nature article, Five Liters is thrilled to move forward with its mission.

Global Von Willebrand Disease (VWD) Treatment Market Report 2023: Increasing Public Awareness Related to VWD Drives Growth - ResearchAndMarkets.com

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Monday, June 12, 2023

On the basis of type of VWD, von Willebrand disease treatment market can be segmented as Type 1 VWD, Type 2 VWD and Type 3 VWD.

Key Points: 
  • On the basis of type of VWD, von Willebrand disease treatment market can be segmented as Type 1 VWD, Type 2 VWD and Type 3 VWD.
  • The types of therapeutic treatments used against von Willebrand disease are Desmopressin (DDAVP), hormone replacement therapy, contraceptives, anti-fibrinolytic or clot-stabilizing drugs, and fibrin sealants.
  • The key factor responsible for growth of von Willebrand disease is increasing public awareness related to VWD which is fueling the diagnosis rate in North America and Europe.
  • North America was observed as the largest market for von Willebrand disease treatment followed by Europe.

Konica Minolta Partners with Scylla AI to Offer Visible Weapon Detection

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Thursday, June 1, 2023

Konica Minolta’s REACT is a comprehensive, end-to-end platform that helps organizations analyze situations and behaviors, offering advanced on-site (edge-based) real-time recognition and judgment-based AI processing.

Key Points: 
  • Konica Minolta’s REACT is a comprehensive, end-to-end platform that helps organizations analyze situations and behaviors, offering advanced on-site (edge-based) real-time recognition and judgment-based AI processing.
  • With the REACT platform, clients can easily add other modules to their security portfolio, such as the new Visible Weapon Detection module, which leverages technology from Scylla AI , a computer vision company based in Austin, Texas.
  • Working with Scylla AI, Konica Minolta developed an algorithm that can detect visible firearms.
  • “REACT - Visible Weapon Detection primarily focuses on individuals who have a drawn weapon, and it can detect a weapon within a few seconds with a high level of accuracy.

Vega Therapeutics Receives FDA Orphan Drug Designation for VGA039 for the Treatment of von Willebrand Disease

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Tuesday, May 23, 2023

Vega Therapeutics , Inc., a clinical stage biotechnology company developing novel therapies for rare blood disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for VGA039 for the treatment of the rare bleeding disorder, von Willebrand disease (VWD).

Key Points: 
  • Vega Therapeutics , Inc., a clinical stage biotechnology company developing novel therapies for rare blood disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for VGA039 for the treatment of the rare bleeding disorder, von Willebrand disease (VWD).
  • By promoting thrombin generation through targeting Protein S, VGA039 addresses a fundamental mechanism of clot formation in VWD and, as a subcutaneously self-administered antibody therapy, has potential to transform VWD treatment.
  • “Receiving orphan drug designation status is an important step for VGA039,” said Gary Patou, MD, Chief Medical Officer of Vega Therapeutics.
  • In preclinical studies, VGA039 has demonstrated efficacy in VWD, as well as in numerous congenital bleeding disorders.

Vega Therapeutics Initiates Clinical Trial Program for VGA039, a First‑in‑Class Antibody Therapy for von Willebrand Disease

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Thursday, April 6, 2023

Vega Therapeutics , Inc., a clinical stage biotechnology company developing novel therapies for rare blood disorders, today announced that its clinical trial program for VGA039 has been initiated.

Key Points: 
  • Vega Therapeutics , Inc., a clinical stage biotechnology company developing novel therapies for rare blood disorders, today announced that its clinical trial program for VGA039 has been initiated.
  • The clinical trial program will begin with enrollment of healthy volunteers and expand to include patients with von Willebrand disease (VWD).
  • By promoting thrombin generation through targeting Protein S, VGA039 addresses a fundamental mechanism of clot formation in VWD and, as a subcutaneously self-administered antibody therapy, has potential to transform VWD treatment.
  • Vega plans to enroll the Phase 1 clinical trial for VGA039 at clinical trial sites across the U.S. and other countries.