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Thrombotic thrombocytopenic purpura (TTP) is a blood disorder that results in blood clots forming in small blood vessels throughout the body. This results in a low platelet count, low red blood cells due to their breakdown, and often kidney, heart, and brain dysfunction. Symptoms may include large bruises, fever, weakness, shortness of breath, confusion, and headache. Repeated episodes may occur.
Rituximab, sold under the brand name Rituxan among others, is a monoclonal antibody medication used to treat certain autoimmune diseases and types of cancer. It is used for non-Hodgkin lymphoma, chronic lymphocytic leukemia, rheumatoid arthritis, granulomatosis with polyangiitis, idiopathic thrombocytopenic purpura, pemphigus vulgaris, myasthenia gravis and Epstein–Barr virus-positive mucocutaneous ulcers. It is given by slow intravenous infusion. Biosimilars of Rituxan include Blitzima, Riabni, Ritemvia, Rituenza, Rixathon, Ruxience, and Truxima.
ADAMTS13 —also known as von Willebrand factor-cleaving protease (VWFCP)—is a zinc-containing metalloprotease enzyme that cleaves von Willebrand factor (vWf), a large protein involved in blood clotting. It is secreted into the blood and degrades large vWf multimers, decreasing their activity, hence ADAMTS13 acts to reduce thrombus formation.
Thrombotic microangiopathy (TMA) is a pathology that results in thrombosis in capillaries and arterioles, due to an endothelial injury. It may be seen in association with thrombocytopenia, anemia, purpura and kidney failure.
Ocrelizumab, sold under the brand name Ocrevus, is a medication used for the treatment of multiple sclerosis (MS). It is a humanized anti-CD20 monoclonal antibody. It targets CD20 marker on B lymphocytes and is an immunosuppressive drug. Ocrelizumab binds to an epitope that overlaps with the epitope to which rituximab binds.
Eltrombopag, sold under the brand name Promacta among others, is a medication used to treat thrombocytopenia and severe aplastic anemia. Eltrombopag is sold under the brand name Revolade outside the US and is marketed by Novartis. It is a thrombopoietin receptor agonist. It is taken by mouth.
Fostamatinib, sold under the brand names Tavalisse and Tavlesse, is a tyrosine kinase inhibitor medication for the treatment of chronic immune thrombocytopenia (ITP). The drug is administered by mouth.
Upshaw–Schulman syndrome (USS) is the recessively inherited form of thrombotic thrombocytopenic purpura (TTP), a rare and complex blood coagulation disease. USS is caused by the absence of the ADAMTS13 protease resulting in the persistence of ultra large von Willebrand factor multimers (ULVWF), causing episodes of acute thrombotic microangiopathy with disseminated multiple small vessel obstructions. These obstructions deprive downstream tissues from blood and oxygen, which can result in tissue damage and death. The presentation of an acute USS episode is variable but usually associated with thrombocytopenia, microangiopathic hemolytic anemia (MAHA) with schistocytes on the peripheral blood smear, fever and signs of ischemic organ damage in the brain, kidney and heart.
Enfortumab vedotin, sold under the brand name Padcev, is an antibody-drug conjugate used for the treatment of urothelial cancer. It is a nectin-4-directed antibody and microtubule inhibitor conjugate. Enfortumab refers to the monoclonal antibody part, and vedotin refers to the payload drug (MMAE) and the linker.
Polatuzumab vedotin, sold under the brand name Polivy, is a CD79b-directed antibody-drug conjugate medication used for the treatment of diffuse large B-cell lymphoma (cancer). It was developed by the Genentech subsidiary of Roche.
Inebilizumab, sold under the brand name Uplizna, is a medication for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adults. Inebilizumab is a humanized mAb that binds to and depletes CD19+ B cells including plasmablasts and plasma cells.
Galcanezumab, sold under the brand name Emgality, is a humanized monoclonal antibody used for the prevention of migraine. It is also used for cluster headaches.
Ravulizumab, sold under the brand name Ultomiris, is a humanized monoclonal antibody complement inhibitor medication designed for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome. It is designed to bind to and prevent the activation of Complement component 5 (C5).
Satralizumab, sold under the brand name Enspryng, is a humanized monoclonal antibody medication that is used for the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune disease. The drug is being developed by Chugai Pharmaceutical, a subsidiary of Roche.
Sutimlimab, sold under the brand name Enjaymo, is a monoclonal antibody that is used to treat adults with cold agglutinin disease (CAD). It is given by intravenous infusion. Sutimlimab prevents complement-enhanced activation of autoimmune human B cells in vitro.
Tafasitamab, sold under the brand name Monjuvi, is a medication used in combination with lenalidomide for the treatment of adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL).
Idecabtagene vicleucel, sold under the brand name Abecma, is a cell-based gene therapy to treat multiple myeloma.
Vilobelimab, sold under the brand name Gohibic, is an investigational medication that is used for the treatment of COVID-19. It is a human-mouse chimeric IgG4 kappa antibody that targets human C5a in plasma.
Pozelimab, sold under the brand name Veopoz, is a recombinant monoclonal antibody used for the treatment of CD55-deficient protein-losing enteropathy, also known as CHAPLE disease. Pozelimab is a complement inhibitor. It is produced using recombinant DNA technology in Chinese hamster ovary cells.
Apadamtase alfa, sold under the brand name Adzynma, is an enzyme replacement therapy used for the treatment of thrombotic thrombocytopenic purpura. Apadamtase alfa is a human recombinant a disintegrin and metalloproteinase with thrombospondin motifs 13. It is given by injection into a vein.
References
- ↑ "Cablivi Product information". Health Canada. 25 April 2012. Retrieved 29 May 2022.
- ↑ "Summary Basis of Decision (SBD) for Cablivi". Health Canada. 23 October 2014. Retrieved 29 May 2022.
- ↑ "Cablivi 10 mg powder and solvent for solution for injection - Summary of Product Characteristics (SmPC)". (emc). 22 June 2020. Retrieved 22 August 2020.
- ↑ Statement On A Nonproprietary Name Adopted By The USAN Council - Caplacizumab, American Medical Association .
- ↑ World Health Organization (WHO) (2011). "International Nonproprietary Names for Pharmaceutical Substances (INN). Proposed INN: List 106" (PDF). WHO Drug Information. 25 (4).
- ↑ Gómez-Seguí I, Fernández-Zarzoso M, de la Rubia J (November 2020). "A critical evaluation of caplacizumab for the treatment of acquired thrombotic thrombocytopenic purpura". Expert Review of Hematology. 13 (11): 1153–1164. doi:10.1080/17474086.2020.1819230. PMID 32876503. S2CID 221468324.
- ↑ Poullin P, Bornet C, Veyradier A, Coppo P (June 2019). "Caplacizumab to treat immune-mediated thrombotic thrombocytopenic purpura". Drugs of Today. Barcelona, Spain. 55 (6): 367–376. doi:10.1358/dot.2019.55.6.2989843. PMID 31250841. S2CID 195761938.
- ↑ Clinical trial number NCT02553317 for "A Trial With Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura (HERCULES)" at ClinicalTrials.gov
- ↑ "Cablivi EPAR". European Medicines Agency (EMA). Retrieved 7 September 2020.
- 1 2 3 Immune Drug Tackles Microvascular Thrombosis Disorder. February 2016
- ↑ FDA Professional Drug Information
- ↑ "FDA approves first therapy for the treatment of adult patients with a rare blood clotting disorder". U.S. Food and Drug Administration (FDA) (Press release). 6 February 2019. Archived from the original on 23 November 2019. Retrieved 22 November 2019.
This article incorporates text from this source, which is in the public domain . - ↑ "Drug Trials Snapshots: Cablivi". U.S. Food and Drug Administration (FDA). 11 February 2019. Archived from the original on 23 November 2019. Retrieved 22 November 2019. This article incorporates text from this source, which is in the public domain .
- ↑ "New Drug Therapy Approvals 2019". U.S. Food and Drug Administration. 31 December 2019. Retrieved 15 September 2020.
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