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Von Willebrand disease (VWD) is the most common hereditary blood-clotting disorder in humans. An acquired form can sometimes result from other medical conditions. It arises from a deficiency in the quality or quantity of von Willebrand factor (VWF), a multimeric protein that is required for platelet adhesion. It is known to affect several breeds of dogs as well as humans. The three forms of VWD are hereditary, acquired, and pseudo or platelet type. The three types of hereditary VWD are VWD type 1, VWD type 2, and VWD type 3. Type 2 contains various subtypes. Platelet type VWD is also an inherited condition.
Enoxaparin sodium, sold under the brand name Lovenox among others, is an anticoagulant medication. It is used to treat and prevent deep vein thrombosis (DVT) and pulmonary embolism (PE) including during pregnancy and following certain types of surgery. It is also used in those with acute coronary syndrome (ACS) and heart attacks. It is given by injection just under the skin or into a vein. It is also used during hemodialysis.
Defibrotide, sold under the brand name Defitelio, is a mixture of single-stranded oligonucleotides that is purified from the intestinal mucosa of pigs. It is used to treat veno-occlusive disease of the liver of people having had a bone marrow transplant, with different limitations in the US and the European Union. It works by protecting the cells lining blood vessels in the liver and preventing blood clotting; the way it does this is not well understood.
Factor VIII is a medication used to treat and prevent bleeding in people with hemophilia A and other causes of low factor VIII. Certain preparations may also be used in those with von Willebrand's disease. It is given by slow injection into a vein.
Moroctocog alfa is a recombinant antihemophilic factor genetically engineered from Chinese hamster ovary (CHO) cell line. Chemically it is a glycoprotein. It is manufactured by Genetics Institute, Inc. and used to control and prevent hemorrhagic bleeding and prophylaxis associated with surgery or to reduce the number of spontaneous bleeding episodes in patients with hemophilia A. It is partially a recombinant coagulation factor VIII since it has an amino acid sequence which compares to the 90 + 80 kDa form of factor VIII (BDDrFVIII). It also has posttranslational modifications which are similar to those of the plasma-derived molecule. It can not prevent hemorrhagic bleeding associated with von Willebrand's disease since it is not a von Willebrand factor.
Edoxaban, sold under the brand name Lixiana among others, is an anticoagulant medication and a direct factor Xa inhibitor. It is taken by mouth.
Cabozantinib, sold under the brand names Cometriq and Cabometyx among others, is an anti-cancer medication used to treat medullary thyroid cancer, renal cell carcinoma, and hepatocellular carcinoma. It is a small molecule inhibitor of the tyrosine kinases c-Met and VEGFR2, and also inhibits AXL and RET. It was discovered and developed by Exelixis Inc.
Apixaban, sold under the brand name Eliquis, is an anticoagulant medication used to treat and prevent blood clots and to prevent stroke in people with nonvalvular atrial fibrillation through directly inhibiting factor Xa. Specifically, it is used to prevent blood clots following hip or knee replacement and in those with a history of prior clots. It is used as an alternative to warfarin and does not require monitoring by blood tests or dietary restrictions. It is taken by mouth.
Recombinant factor VIIa, also known as eptacog alfa (INN), and sold under the brand name Novoseven, among others, is a form of blood factor VII that has been manufactured via recombinant technology. It is administered via an injection into a vein.
Caplacizumab is a bivalent single-domain antibody (VHH) designed for the treatment of thrombotic thrombocytopenic purpura (TTP) and thrombosis.
Turoctocog alfa is a recombinant antihemophilic factor VIII used for the treatment of and prophylaxis of bleeding patients with haemophilia A. It is marketed by Novo Nordisk. It was approved in the United States, the European Union, and Japan in 2013.
Asfotase alfa, sold under the brand name Strensiq, is a medication used in the treatment of people with perinatal/infantile- and juvenile-onset hypophosphatasia.
Migalastat, sold under the brand name Galafold, is a medication for the treatment of Fabry disease, a rare genetic disorder. It was developed by Amicus Therapeutics. The US Food and Drug Administration (FDA) granted it orphan drug status in 2004, and the European Commission followed in 2006. The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) granted the drug a marketing approval under the name Galafold in May 2016.
Insulin degludec/liraglutide, sold under the brand name Xultophy, is a fixed-dose combination medication for the treatment of adults with type 2 diabetes to improve glycemic control in combination with diet and exercise. It contains insulin degludec and liraglutide. It is administered by subcutaneous injection.
Luspatercept, sold under the brand name Reblozyl, is a medication used for the treatment of anemia in beta thalassemia and myelodysplastic syndromes.
Trastuzumab deruxtecan, sold under the brand name Enhertu, is an antibody-drug conjugate consisting of the humanized monoclonal antibody trastuzumab (Herceptin) covalently linked to the topoisomerase I inhibitor deruxtecan. It is licensed for the treatment of breast cancer or gastric or gastroesophageal adenocarcinoma. Trastuzumab binds to and blocks signaling through epidermal growth factor receptor 2 (HER2/neu) on cancers that rely on it for growth. Additionally, once bound to HER2 receptors, the antibody is internalized by the cell, carrying the bound deruxtecan along with it, where it interferes with the cell's ability to make DNA structural changes and replicate its DNA during cell division, leading to DNA damage when the cell attempts to replicate itself, destroying the cell.
Susoctocog alfa, sold under the brand name Obizur, is a medication used for the treatment of bleeding episodes in adults with acquired haemophilia, a bleeding disorder caused by the spontaneous development of antibodies that inactivate factor VIII.
Efmoroctocog alfa, sold under the brand name Elocta among others, is a medication for the treatment and prophylaxis of bleeding in people with hemophilia A. Efmoroctocog alfa is a recombinant human coagulation factor VIII, Fc fusion protein (rFVIIIFc). It is produced by recombinant DNA technology in a human embryonic kidney (HEK) cell line.
Damoctocog alfa pegol, sold under the brand name Jivi is a recombinant DNA-derived, Factor VIII concentrate medication used to treat hemophilia A.
Avalglucosidase alfa, sold under the brand name Nexviazyme, is an enzyme replacement therapy medication used for the treatment of glycogen storage disease type II.
References
- 1 2 3 "Veyvondi Australian prescription medicine decision summary". Therapeutic Goods Administration (TGA). 29 April 2020. Retrieved 16 August 2020.
- ↑ "Summary Basis of Decision (SBD) for Vonvendi". Health Canada . 23 October 2014. Retrieved 29 May 2022.
- ↑ "Vonvendi 650 IU powder and solvent for solution for injection - Summary of Product Characteristics (SmPC)". (emc). 7 May 2020. Retrieved 16 August 2020.
- 1 2 "Vonvendi". U.S. Food and Drug Administration (FDA). 9 May 2018. Archived from the original on 23 April 2019. Retrieved 15 April 2020.
{{cite web}}: CS1 maint: unfit URL (link) - 1 2 3 4 5 6 7 8 9 10 "Veyvondi EPAR". European Medicines Agency (EMA). 20 September 2018. Retrieved 27 March 2020. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- 1 2 3 4 "Vonvendi (von willebrand factor- recombinant kit". DailyMed. 13 February 2019. Retrieved 27 March 2020.
- ↑ "Veyvondi-epar product information" (PDF). European Medicines Agency.
- ↑ Tran T, Arnall J, Moore DC, Ward L, Palkimas S, Man L (April 2020). "Vonicog alfa for the management of von Willebrand disease: a comprehensive review and single-center experience". Journal of Thrombosis and Thrombolysis. 49 (3): 431–440. doi:10.1007/s11239-019-02018-1. PMID 31902122. S2CID 209673044.
- ↑ "Vonicog alfa". Specialist Pharmacy Service. 15 January 2020. Archived from the original on 27 March 2020. Retrieved 27 March 2020.
- ↑ "Vonvendi". U.S. Food and Drug Administration (FDA). 13 April 2018. STN: 125577. Retrieved 27 March 2020.
