Eltrombopag

Eltrombopag is a TPO-receptor agonist that interacts with the transmembrane domain of the human TPO-receptor (also known as cMpl) and initiates signaling cascades that induce proliferation and differentiation of megakaryocytes leading to increased platelet production.

• Eltrombopag is a thrombopoietin receptor agonist indicated: for the treatment of thrombocytopenia in adult and pediatric patients 1 year and older with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. Eltrombopag tablets should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding.

• None. None.contraindicated

Take eltrombopag tablets without a meal or with a meal low in calcium (≤ 50 mg). Take eltrombopag tablets at least 2 hours before or 4 hours after any medications or products containing polyvalent cations, such as antacids, calcium-rich foods, and mineral supplements. ( 2.4 , 7.1 , 12.3 ) Persistent or Chronic ITP: Initiate eltrombopag tablets at 50 mg orally once daily for most adult and pediatric patients 6 years and older, and at 25 mg orally once daily for pediatric patients aged 1 to 5 years. Dose reductions are needed for patients with hepatic impairment and some patients of East-/Southeast-Asian ancestry. Adjust to maintain platelet count greater than or equal to 50 x 10 9 /L. Do not exceed 75 mg per day. ( 2.1 , 8.6 , 8.7 ) Chronic Hepatitis C-associated Thrombocytopenia: Initiate eltrombopag tablets at 25 mg orally once daily for all patients. Adjust to achieve target platelet count required to initiate antiviral therapy. Do not exceed a daily dose of 100 mg. ( 2.2 ) First-line Severe Aplastic Anemia: Initiate eltrombopag tablets orally once daily at 2.5 mg/kg (in pediatric patients aged 2 to 5 years old), 75 mg (pediatric patients aged 6 to 11 years old) or 150 mg for patients aged 12 years and older concurrently with standard immunosuppressive therapy. Reduce initial dose in patients of East-/Southeast-Asian ancestry. Modify dosage for toxicity or elevated platelet counts. ( 2.3 , 8.7 ) Refractory Severe Aplastic Anemia: Initiate eltrombopag tablets orally at 50 mg once daily.

Hepatotoxicity: Monitor liver function before and during therapy. ( 5.2 ) Increased Risk of Death and Progression of Myelodysplastic Syndromes to Acute Myeloid Leukemia. ( 5.3 ) Thrombotic/Thromboembolic Complications: Portal vein thrombosis has been reported in patients with chronic liver disease receiving eltrombopag. Monitor platelet counts regularly. ( 5.4 ) 5.1 Hepatic Decompensation in Patients With Chronic Hepatitis C In patients with chronic hepatitis C, eltrombopag in combination with interferon and ribavirin may increase the risk of hepatic decompensation. In two controlled clinical trials in patients with chronic hepatitis C and thrombocytopenia, ascites and encephalopathy occurred more frequently on the arm receiving treatment with eltrombopag plus antivirals (7%) than the placebo plus antivirals arm (4%). Patients with low albumin levels (less than 3.5 g/dL) or Model for End-Stage Liver Disease (MELD) score greater than or equal to 10 at baseline had a greater risk for hepatic decompensation on the arm receiving treatment with eltrombopag plus antivirals. Discontinue eltrombopag if antiviral therapy is discontinued. 5.2 Hepatotoxicity Eltrombopag may increase the risk of severe and potentially life-threatening hepatotoxicity [see Adverse Reactions (6.1) ] . One patient (< 1%) with ITP treated with eltrombopag in clinical trials experienced drug-induced liver injury. Eleven patients (1%) with chronic hepatitis C treated with eltrombopag in clinical trials experienced drug-induced liver injury.

The following clinically significant adverse reactions associated with eltrombopag are described in other sections. Hepatic Decompensation in Patients with Chronic Hepatitis C [see Warnings and Precautions (5.1) ] Hepatotoxicity [see Warnings and Precautions (5.2) ] Increased Risk of Death and Progression of Myelodysplastic Syndromes to Acute Myeloid Leukemia [see Warnings and Precautions (5.3) ] Thrombotic/Thromboembolic Complications [see Warnings and Precautions (5.4) ] Cataracts [see Warnings and Precautions (5.5) ] Across all indications, the most common adverse reactions (≥ 20% in any indication) were: anemia, nausea, pyrexia, alanine aminotransferase increased, cough, fatigue, headache, and diarrhea. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Amneal Pharmaceuticals LLC at 1-877-835-5472 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared with rates in the clinical trials of another drug and may not reflect the rates observed in practice. Persistent or Chronic Immune Thrombocytopenia Adults In clinical trials, hemorrhage was the most common serious adverse reaction and most hemorrhagic reactions followed discontinuation of eltrombopag. Other serious adverse reactions included thrombotic/thromboembolic complications [see Warnings and Precautions (5.4) ] .

Lactation: Advise women not to breastfeed during treatment. ( 8.2 ) 8.1 Pregnancy Risk Summary Available data from a small number of published case reports and post-marketing experience with eltrombopag use in pregnant women are insufficient to assess any drug-associated risks for major birth defects, miscarriage, or adverse maternal or fetal outcomes. In animal reproduction and developmental toxicity studies, oral administration of eltrombopag to pregnant rats during organogenesis resulted in embryolethality and reduced fetal weights at maternally toxic doses. These effects were observed at doses resulting in exposures that were six times the human clinical exposure based on area under the curve (AUC) in patients with persistent or chronic ITP at 75 mg/day, and three times the AUC in patients with chronic hepatitis C at 100 mg/day (see Data) . The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and of miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.

Metabolism

Eltrombopag demonstrated a dose-proportional increase in exposure between doses of 50 to 150 mg/day in healthy adult subjects. Eltrombopag AUC was approximately 1.7-fold higher in patients with persistent or chronic ITP and approximately 2.8-fold higher in patients with HCV compared to healthy subjects.

In the event of overdose, platelet counts may increase excessively and result in thrombotic/thromboembolic complications. In one report, a subject who ingested 5,000 mg of eltrombopag had a platelet count increase to a maximum of 929 x 10 9 /L at 13 days following the ingestion. The patient also experienced rash, bradycardia, ALT/AST elevations, and fatigue. The patient was treated with gastric lavage, oral lactulose, intravenous fluids, omeprazole, atropine, furosemide, calcium, dexamethasone, and plasmapheresis; however, the abnormal platelet count and liver test abnormalities persisted for 3 weeks. After 2 months’ follow-up, all events had resolved without sequelae. In case of an overdose, consider oral administration of a metal cation-containing preparation, such as calcium, aluminum, or magnesium preparations to chelate eltrombopag and thus limit absorption. Closely monitor platelet counts. Reinitiate treatment with eltrombopag in accordance with dosing and administration recommendations [see Dosage and Administration (2.1 , 2.2) ] . Consider contacting the Poison Help line (1­-800-222-1222) or a medical toxicologist for additional overdose management recommendations.

• Nov 20, 2008NDANDA022291Novartis
• Aug 24, 2015NDANDA207027Novartis
• Nov 29, 2023NDANDA216774Teva Pharms Inc
• Apr 18, 2024ANDAANDA216620Annora Pharma
• Jan 17, 2025ANDAANDA206788Hetero Labs Ltd V
• Jan 14, 2026ANDAANDA219638Somerset Theraps Llc
• Jan 14, 2026ANDAANDA212884Amneal
• Jan 14, 2026ANDAANDA220250Msn

1. 1Platelet Count Decreased3,97018%
2. 2Death3,31615%
3. 3Drug Ineffective1,9468.6%
4. 4Fatigue1,1114.9%
5. 5Platelet Count Increased1,1074.9%
6. 6Headache9694.3%
7. 7Off Label Use8903.9%
8. 8Nausea8093.6%
9. 9Diarrhoea8033.5%
10. 10Product Use In Unapproved Indication7403.3%
11. 11Malaise5862.6%
12. 12Thrombocytopenia5822.6%
13. 13Pyrexia5472.4%
14. 14Asthenia5332.4%
15. 15Contusion5082.2%

The 10 most recently updated of 196 ClinicalTrials.gov registrations naming Eltrombopag as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
  Recruiting · Phase 2 · Interventional · 164 enrolled · Novartis Pharmaceuticals
  NCT07421167updated 2026-06-11
• Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
  Enrolling by invitation · Phase 1 · Phase 2 · Interventional · 10 enrolled · National Institute of Allergy and Infectious Diseases (NIAID)
  NCT07113743updated 2026-06-08
• Eltrombopag as a Novel Therapeutic Approach for Low-risk MDS and CMML With TET2 Mutations
  Suspended · Phase 2 · Interventional · 25 enrolled · Abhay Singh, MD MPH
  NCT06630221updated 2026-06-05
• CsA+EPAG/HPAG+Romiplostim N01 in the Treatment of Newly-diagnosed SAA/TD-NSAA
  Withdrawn · Phase 2 · Interventional · 0 enrolled · Peking Union Medical College Hospital
  NCT07523009updated 2026-06-04
• A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids
  Active not recruiting · Phase 3 · Interventional · 152 enrolled · Novartis Pharmaceuticals
  NCT05653219updated 2026-05-27
• A Real-world Study of Treatment Patterns and Outcomes in Primary Immune Thrombocytopenia Patients Treated With Eltrombopag
  Not yet recruiting · Observational · 150 enrolled · Novartis Pharmaceuticals
  NCT07587723updated 2026-05-14
• CsA+EPAG/HPAG+Romiplostim N01 in Newly-diagnosed SAA/TD-NSAA
  Not yet recruiting · Phase 2 · Interventional · 43 enrolled · Peking Union Medical College Hospital
  NCT07558434updated 2026-04-30
• Eltrombopag Combining Rituximab Versus Eltrombopag in the Management of Primary Immune Thrombocytopenia (ITP) in Adults
  Recruiting · Phase 4 · Interventional · 224 enrolled · Institute of Hematology & Blood Diseases Hospital, China
  NCT04518475updated 2026-04-29
• rhTPO Dose Escalation vs Eltrombopag Switch in ITP
  Not yet recruiting · Interventional · 112 enrolled · Institute of Hematology & Blood Diseases Hospital, China
  NCT07476846updated 2026-04-27
• hATG+CsA vs hATG+CsA+Eltrombopag for SAA
  Completed · Phase 3 · Interventional · 202 enrolled · European Society for Blood and Marrow Transplantation
  NCT02099747updated 2026-04-24

CPIC-curated drug–gene pairs for Eltrombopag. Levels describe the strength of curated evidence and guideline status — never a recommendation to test or to adjust therapy.

• F5CPIC C (provisional)FDA label: Actionable PGx
• SERPINC1CPIC D (provisional)FDA label: Actionable PGx

Frequently asked questions

How does Eltrombopag work?

Eltrombopag is a TPO-receptor agonist that interacts with the transmembrane domain of the human TPO-receptor (also known as cMpl) and initiates signaling cascades that induce proliferation and differentiation of megakaryocytes leading to increased platelet production.

What is Eltrombopag used for?

According to FDA labeling, Eltrombopag carries indications including: Eltrombopag is a thrombopoietin receptor agonist indicated: for the treatment of thrombocytopenia in adult and pediatric patients 1 year and older with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. Eltrombopag tablets should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Eltrombopag?

Eltrombopag is classified as Other systemic hemostatics, Thrombopoietin Receptor Agonist, Breast Cancer Resistance Protein Inhibitors, Organic Anion Transporting Polypeptide 1B1 Inhibitors, Thrombopoietin Receptor Agonists, UGT1A1 Inhibitors, UGT1A3 Inhibitors, UGT1A4 Inhibitors, UGT1A6 Inhibitors, UGT1A9 Inhibitors, UGT2B15 Inhibitors, UGT2B7 Inhibitors, Increased Megakaryocyte Maturation, Increased Platelet Production.

What are the brand names for Eltrombopag?

Eltrombopag is marketed under brand names including Alvaiz, Promacta.

What are the contraindications for Eltrombopag?

Eltrombopag labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.