- Marketing Authorization will enable patient access to VEYVONDI® throughout Europe
- VEYVONDI [vonicog alfa, recombinant von Willebrand factor] is the first and only recombinant von Willebrand Factor (rVWF) to treat hemorrhage and treat/prevent surgical bleeding in adults (age 18 and older) with von Willebrand disease (VWD)1
ZUG, Switzerland I September 12, 2018 I Shire plc (LSE: SHP, NASDAQ: SHPG) the global biotech leader in rare diseases, announced today that the European Commission (EC) has granted Marketing Authorization for VEYVONDI [vonicog alfa, recombinant von Willebrand factor] (rVWF), for the treatment of bleeding events and treatment/prevention of surgical bleeding in adults (age 18 and older) with von Willebrand disease (VWD) when desmopressin (DDAVP) treatment alone is ineffective or not indicated. VEYVONDI should not be used in the treatment of hemophilia A.1 VEYVONDI is the first and only recombinant von Willebrand Factor (rVWF) treatment in the EU for von Willebrand disease (VWD) that specifically addresses the primary deficiency or dysfunction of von Willebrand Factor (VWF) while also allowing the body to restore and maintain adequate Factor VIII (FVIII) plasma levels.1
“The approval in Europe for VEYVONDI marks a key milestone in our efforts to tackle unmet medical needs for those living with von Willebrand disease,” said Andreas Busch, Head of Research and Development and Chief Scientific Officer, Shire. “We are excited to take the next steps in ensuring that VEYVONDI is widely available across Europe to address the individual needs of those affected by the condition and in need of factor replacement.”
The Marketing Authorization is based on outcomes from three clinical trials of a total 80 patients with VWD exposed to VEYVONDI. These include a Phase 1 multicenter, controlled, randomized, single-blind, dose-escalation study of the safety, tolerability and pharmacokinectics (PK) of rVWF:rFVIII in subjects 18 to 60 years of age with severe VWD; a Phase 3 multicenter, open-label study to assess the PK, safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding episodes in adult subjects with severe VWD; and a Phase 3, prospective, open-label, uncontrolled, non-randomized, international multicenter study to assess the hemostatic efficacy and safety of rVWF with or without rFVIII in 15 adult subjects with severe VWD undergoing major, minor, or oral elective surgical procedures.2
VWD is the most common inherited bleeding disorder, affecting up to 1 percent of the global population or approximately 100,000 people in the EU.3,4 VWD is caused by a deficiency or dysfunction of VWF, one of several types of proteins in the blood that are needed to facilitate proper blood clotting.5 Only a minor proportion of affected individuals have the severe form of the disease and are in need of VWF replacement.6 Symptoms range from nosebleeds to bleeding from the gums and easy bruising. Bleeding from the stomach and intestines can occur but is less common.7
With this approval, Shire is now authorized to market VEYVONDI in the 28 Member States of the European Union (EU), as well as in Iceland, Lichtenstein and Norway.
About von Willebrand disease (VWD)
VWD is the most common inherited bleeding disorder, affecting up to 1 percent of the global population.3 VWD is caused by a deficiency or dysfunction of VWF, one of several types of proteins in the blood that are needed to facilitate proper blood clotting.5 Due to this deficiency or dysfunction in VWF, blood is not able to clot effectively in people with VWD.6 The majority of cases (70-80%) are due to partial quantitative VWF deficiency (Type 1 VWD).8 The most severe form of VWD (severe Type 3 VWD) is the least common, with incidence varying by ethnicity from 0.1 to 5.3 cases per million.9 The mutation may occur spontaneously with no previous family history.7 Bleeding caused by VWD is unpredictable and its severity varies greatly among patients with this disease.10
About VEYVONDI
VEYVONDI is indicated in adults (age 18 and older) with von Willebrand disease (VWD), when desmopressin (DDAVP) treatment alone is ineffective or not indicated for the treatment of hemorrhage and treatment/prevention of surgical bleeding. VEYVONDI should not be used in the treatment of hemophilia A.1
For full EU Summary of Product Characteristics, including approved indication(s) and important safety information about marketed products, please visit: http://ec.europa.eu/health/documents/community-register/2018/20180831141871/anx_141871_en.pdf1
In the US the product has been approved for more than two years under the trade name VONVENDI® [von Willebrand factor (Recombinant)] and is indicated for use in adults (age 18 and older) diagnosed with von Willebrand disease for on-demand treatment and control of bleeding episodes or perioperative management of bleeding.
About Shire
Shire is the global biotechnology leader serving patients with rare diseases and specialized conditions. We seek to push boundaries through discovering and delivering new possibilities for patient communities who often have few or no other champions. Relentlessly on the edge of what’s next, we are serial innovators with a diverse pipeline offering fresh thinking and new hope. Serving patients and partnering with healthcare communities in over 100 countries, we strive to be part of the entire patient journey to enable earlier diagnosis, raise standards of care, accelerate access to treatment, and support patients. Our diverse portfolio of therapeutic areas includes Immunology, Hematology, Genetic Diseases, Neuroscience, Internal Medicine, and Ophthalmics.
REFERENCES
1 European Commission. Veyvondi Summary of Product Characteristics. Available at: http://ec.europa.eu/health/documents/community-register/2018/20180831141871/anx_141871_en.pdf
2 Recombinant von Willebrand Factor; rVWF; vonicog alfa; BAX 111. Clinical Overview. May 2017.
3 World Federation of Hemophilia. What is von Willebrand disease (VWD)? Available at: www.wfh.org/en/page.aspx?pid=673.
4 European Medicines Agency. EU/3/10/814. Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/orphans/2010/12/human_orphan_000865.jsp&mid=WC0b01ac058001d12b.
5 National Hemophilia Foundation. Von Willebrand Disease. Available at: https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Von-Willebrand-Disease.
6 National Organization for Rare Disorders. Von Willebrand Disease. Available at: https://rarediseases.org/rare-diseases/von-willebrand-disease/.
7 Rare Diseases. Von Willebrand Disease. Available at: https://rarediseases.org/rare-diseases/von-willebrand-disease/.
8 Kujovic, J.L. Von Willebrand’s Disease and Menorrhagia: Prevalence, Diagnosis, and Management. Available at: https://onlinelibrary.wiley.com/doi/pdf/10.1002/ajh.20372.
9 Verma, A., Yaish, H. et al. Type 3 Von Willebrand Disease with Alloantibodies and Its Challenging Management During Episode of Bleeding. Available at: http://www.bloodjournal.org/content/116/21/1405.
10 National Hemophilia Foundation. “VWD Summit Highlights.” HEMAWARE website. https://hemaware.org/story/vwd-summit-highights.
SOURCE: Shire
Post Views: 45
- Marketing Authorization will enable patient access to VEYVONDI® throughout Europe
- VEYVONDI [vonicog alfa, recombinant von Willebrand factor] is the first and only recombinant von Willebrand Factor (rVWF) to treat hemorrhage and treat/prevent surgical bleeding in adults (age 18 and older) with von Willebrand disease (VWD)1
ZUG, Switzerland I September 12, 2018 I Shire plc (LSE: SHP, NASDAQ: SHPG) the global biotech leader in rare diseases, announced today that the European Commission (EC) has granted Marketing Authorization for VEYVONDI [vonicog alfa, recombinant von Willebrand factor] (rVWF), for the treatment of bleeding events and treatment/prevention of surgical bleeding in adults (age 18 and older) with von Willebrand disease (VWD) when desmopressin (DDAVP) treatment alone is ineffective or not indicated. VEYVONDI should not be used in the treatment of hemophilia A.1 VEYVONDI is the first and only recombinant von Willebrand Factor (rVWF) treatment in the EU for von Willebrand disease (VWD) that specifically addresses the primary deficiency or dysfunction of von Willebrand Factor (VWF) while also allowing the body to restore and maintain adequate Factor VIII (FVIII) plasma levels.1
“The approval in Europe for VEYVONDI marks a key milestone in our efforts to tackle unmet medical needs for those living with von Willebrand disease,” said Andreas Busch, Head of Research and Development and Chief Scientific Officer, Shire. “We are excited to take the next steps in ensuring that VEYVONDI is widely available across Europe to address the individual needs of those affected by the condition and in need of factor replacement.”
The Marketing Authorization is based on outcomes from three clinical trials of a total 80 patients with VWD exposed to VEYVONDI. These include a Phase 1 multicenter, controlled, randomized, single-blind, dose-escalation study of the safety, tolerability and pharmacokinectics (PK) of rVWF:rFVIII in subjects 18 to 60 years of age with severe VWD; a Phase 3 multicenter, open-label study to assess the PK, safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding episodes in adult subjects with severe VWD; and a Phase 3, prospective, open-label, uncontrolled, non-randomized, international multicenter study to assess the hemostatic efficacy and safety of rVWF with or without rFVIII in 15 adult subjects with severe VWD undergoing major, minor, or oral elective surgical procedures.2
VWD is the most common inherited bleeding disorder, affecting up to 1 percent of the global population or approximately 100,000 people in the EU.3,4 VWD is caused by a deficiency or dysfunction of VWF, one of several types of proteins in the blood that are needed to facilitate proper blood clotting.5 Only a minor proportion of affected individuals have the severe form of the disease and are in need of VWF replacement.6 Symptoms range from nosebleeds to bleeding from the gums and easy bruising. Bleeding from the stomach and intestines can occur but is less common.7
With this approval, Shire is now authorized to market VEYVONDI in the 28 Member States of the European Union (EU), as well as in Iceland, Lichtenstein and Norway.
About von Willebrand disease (VWD)
VWD is the most common inherited bleeding disorder, affecting up to 1 percent of the global population.3 VWD is caused by a deficiency or dysfunction of VWF, one of several types of proteins in the blood that are needed to facilitate proper blood clotting.5 Due to this deficiency or dysfunction in VWF, blood is not able to clot effectively in people with VWD.6 The majority of cases (70-80%) are due to partial quantitative VWF deficiency (Type 1 VWD).8 The most severe form of VWD (severe Type 3 VWD) is the least common, with incidence varying by ethnicity from 0.1 to 5.3 cases per million.9 The mutation may occur spontaneously with no previous family history.7 Bleeding caused by VWD is unpredictable and its severity varies greatly among patients with this disease.10
About VEYVONDI
VEYVONDI is indicated in adults (age 18 and older) with von Willebrand disease (VWD), when desmopressin (DDAVP) treatment alone is ineffective or not indicated for the treatment of hemorrhage and treatment/prevention of surgical bleeding. VEYVONDI should not be used in the treatment of hemophilia A.1
For full EU Summary of Product Characteristics, including approved indication(s) and important safety information about marketed products, please visit: http://ec.europa.eu/health/documents/community-register/2018/20180831141871/anx_141871_en.pdf1
In the US the product has been approved for more than two years under the trade name VONVENDI® [von Willebrand factor (Recombinant)] and is indicated for use in adults (age 18 and older) diagnosed with von Willebrand disease for on-demand treatment and control of bleeding episodes or perioperative management of bleeding.
About Shire
Shire is the global biotechnology leader serving patients with rare diseases and specialized conditions. We seek to push boundaries through discovering and delivering new possibilities for patient communities who often have few or no other champions. Relentlessly on the edge of what’s next, we are serial innovators with a diverse pipeline offering fresh thinking and new hope. Serving patients and partnering with healthcare communities in over 100 countries, we strive to be part of the entire patient journey to enable earlier diagnosis, raise standards of care, accelerate access to treatment, and support patients. Our diverse portfolio of therapeutic areas includes Immunology, Hematology, Genetic Diseases, Neuroscience, Internal Medicine, and Ophthalmics.
REFERENCES
1 European Commission. Veyvondi Summary of Product Characteristics. Available at: http://ec.europa.eu/health/documents/community-register/2018/20180831141871/anx_141871_en.pdf
2 Recombinant von Willebrand Factor; rVWF; vonicog alfa; BAX 111. Clinical Overview. May 2017.
3 World Federation of Hemophilia. What is von Willebrand disease (VWD)? Available at: www.wfh.org/en/page.aspx?pid=673.
4 European Medicines Agency. EU/3/10/814. Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/orphans/2010/12/human_orphan_000865.jsp&mid=WC0b01ac058001d12b.
5 National Hemophilia Foundation. Von Willebrand Disease. Available at: https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Von-Willebrand-Disease.
6 National Organization for Rare Disorders. Von Willebrand Disease. Available at: https://rarediseases.org/rare-diseases/von-willebrand-disease/.
7 Rare Diseases. Von Willebrand Disease. Available at: https://rarediseases.org/rare-diseases/von-willebrand-disease/.
8 Kujovic, J.L. Von Willebrand’s Disease and Menorrhagia: Prevalence, Diagnosis, and Management. Available at: https://onlinelibrary.wiley.com/doi/pdf/10.1002/ajh.20372.
9 Verma, A., Yaish, H. et al. Type 3 Von Willebrand Disease with Alloantibodies and Its Challenging Management During Episode of Bleeding. Available at: http://www.bloodjournal.org/content/116/21/1405.
10 National Hemophilia Foundation. “VWD Summit Highlights.” HEMAWARE website. https://hemaware.org/story/vwd-summit-highights.
SOURCE: Shire
Post Views: 45
