Eltrombopag isindicated for the treatment of thrombocytopenia in people with persistent or chronic immune thrombocytopenia who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy;[3] for the treatment of thrombocytopenia in people with chronic hepatitis C to allow the initiation and maintenance of interferon-based therapy;[3] in combination with standard immunosuppressive therapy for the first-line treatment of people with severe aplastic anemia;[3] and for the treatment of people with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy.[3][10]
Inpreclinical studies, the compound was shown to interact selectively with the thrombopoietin receptor, leading to activation of theJAK-STAT signaling pathway and increased proliferation and differentiation ofmegakaryocytes. Animal studies confirmed that it increased platelet counts. In 73 healthy volunteers, higher doses of eltrombopag caused larger increases in the number of circulating platelets without tolerability problems.[11]
After six weeks of therapy in aphase III trial, eltrombopag 50 mg/day was associated with a significantly higher response rate than placebo in adult patients with chronicidiopathic thrombocytopenic purpura (ITP).[14]
In October 2024, theCommittee for Medicinal Products for Human Use of theEuropean Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Eltrombopag Viatris, intended for the treatment of people with primary immune thrombocytopenia (ITP) and thrombocytopenia associated with chronic hepatitis C.[17] The applicant for this medicinal product is Viatris Limited.[17] Eltrombopag Viatris was authorized in December 2024.[18]
It has been shown to produce a trilineage hematopoiesis in some people with aplastic anemia, resulting in increased platelet counts, along with red and white blood cell counts.[19] Eltrombopag has been shown to targetELAVL1/HuR-RNA interactions affectinggene expression,iron metabolism, andglycoprotein hormones, alpha polypeptide (CGA) levels.[20] The transcription factor EB (TFEB) has been detected as an Eltrombopag target in starvation-induced conditions.[21]
^ab"Eltrombopag Viatris EPAR".European Medicines Agency (EMA). 17 October 2024. Retrieved19 October 2024.Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
Clinical trial numberNCT00102739 for "SB-497115 (Oral Thrombopoietin Receptor Agonist) Versus Placebo In Adults With Refractory Immune Thrombocytopenic Purpura (ITP)" atClinicalTrials.gov
Clinical trial numberNCT00370331 for "RAISE: Randomized Placebo-Controlled Idiopathic Thrombocytopenic Purpura (ITP) Study With Eltrombopag (RAISE)" atClinicalTrials.gov
Clinical trial numberNCT01520909 for "Study of a New Medication for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder of Low Platelet Counts That Can Lead to Bruising Easily, Bleeding Gums, and/or Bleeding Inside the Body. (PETIT2)" atClinicalTrials.gov
Clinical trial numberNCT00908037 for "Efficacy and Safety Study of Eltrombopag in Pediatric Patients With Thrombocytopenia From Chronic Idiopathic Thrombocytopenic Purpura (ITP) (PETIT)" atClinicalTrials.gov
Clinical trial numberNCT00516321 for "Eltrombopag To Initiate And Maintain Interferon Antiviral Treatment To Subjects With Hepatitis C Related Liver Disease" atClinicalTrials.gov
Clinical trial numberNCT00529568 for "Eltrombopag To Initiate And Maintain Interferon Antiviral Treatment To Benefit Subjects With Hepatitis C Liver Disease" atClinicalTrials.gov
Clinical trial numberNCT01623167 for "Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia" atClinicalTrials.gov
Clinical trial numberNCT00922883 for "A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients" atClinicalTrials.gov
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