Avatrombopag

Avatrombopag is an orally bioavailable, small molecule TPO receptor agonist that stimulates proliferation and differentiation of megakaryocytes from bone marrow progenitor cells, resulting in an increased production of platelets. Avatrombopag does not compete with TPO for binding to the TPO receptor and has an additive effect with TPO on platelet production.

• DOPTELET is a thrombopoietin receptor agonist indicated for the treatment of: Thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure. ( 1.1 ) Thrombocytopenia in adult patients with chronic immune thrombocytopenia who have had an insufficient response to a previous treatment.

• None. None.contraindicated

DOPTELET tablets and DOPTELET SPRINKLE are not substitutable on a mg-to-mg basis. DOPTELET SPRINKLE capsules should be opened, and the contents (oral granules) mixed with a soft food or liquid. Administer immediately after mixing. Do not swallow the capsules whole. Administer DOPTELET tablets and DOPTELET SPRINKLE with food. ( 2.1 , 2.3 , 2.5 ) Chronic Liver Disease : Dose DOPTELET tablets based upon platelet count prior to procedure, orally for 5 days beginning 10 days to 13 days before procedure. For platelet count less than 40×10 9 /L, the dose is 60 mg (3 tablets) orally once daily; for platelet count 40 to less than 50×10 9 /L the dose is 40 mg (2 tablets) orally once daily. ( 2.2 ) Adult Patients with Chronic Immune Thrombocytopenia and Pediatric Patients 6 Years and Older with Persistent or Chronic Immune Thrombocytopenia : Initiate DOPTELET tablets at 20 mg (1 tablet) orally once daily. Adjust the dose or frequency of dosing to maintain platelet count greater than or equal to 50×10 9 /L. Do not exceed 40 mg (2 tablets) per day. Pediatric Patients 1 Year to Less than 6 Years with Persistent or Chronic Immune Thrombocytopenia : Initiate DOPTELET SPRINKLE oral granules at 10 mg (content of 1 capsule) orally once daily. Adjust the dose or frequency of dosing to maintain platelet count greater than or equal to 50×10 9 /L. Do not exceed 20 mg (content of 2 capsules) per day. 2.1 Important Use and Administration Instructions Select the recommended product (DOPTELET tablets or DOPTELET SPRINKLE) based on the indication and patient’s age.

Thrombotic/Thromboembolic Complications: DOPTELET is a thrombopoietin (TPO) receptor agonist and TPO receptor agonists have been associated with thrombotic and thromboembolic complications in patients with chronic liver disease or immune thrombocytopenia. Monitor platelet counts. Monitor for signs and symptoms of thromboembolic events and institute treatment promptly. ( 5.1 ) 5.1 Thrombotic/Thromboembolic Complications DOPTELET is a thrombopoietin (TPO) receptor agonist and TPO receptor agonists have been associated with thrombotic and thromboembolic complications in patients with chronic liver disease or immune thrombocytopenia. In patients with chronic liver disease, thromboembolic events (portal vein thrombosis) occurred in 0.4% (1/274) of patients receiving DOPTELET. In adult patients with chronic immune thrombocytopenia, thromboembolic events (arterial or venous) occurred in 7% (9/128) of patients receiving DOPTELET. Consider the potential increased thrombotic risk when administering DOPTELET to patients with known risk factors for thromboembolism, including genetic prothrombotic conditions (e.g., Factor V Leiden, Prothrombin 20210A, Antithrombin deficiency or Protein C or S deficiency) and acquired risk factors (e.g., antiphospholipid syndrome). DOPTELET should not be administered to patients with chronic liver disease or immune thrombocytopenia in an attempt to normalize platelet counts. Monitor platelet counts and follow the dosing guidelines to achieve target platelet counts [see Dosage and Administration ( 2.3 and 2.5 )] .

The following clinically significant adverse reactions are discussed in detail in other sections of the labeling: Thrombotic/Thromboembolic Complications [ see Warnings and Precautions ( 5.1 ) ] In adult patients with chronic liver disease, the most common adverse reactions (≥3%) were pyrexia, abdominal pain, nausea, headache, fatigue, and edema peripheral. ( 6.1 ) In adult patients with chronic immune thrombocytopenia, the most common adverse reactions (≥10%) were headache, fatigue, contusion, epistaxis, upper respiratory tract infection, arthralgia, gingival bleeding, petechiae and nasopharyngitis. ( 6.1 ) In pediatric patients with persistent or chronic immune thrombocytopenia, the most common adverse reactions (≥10%) were viral infection, nasopharyngitis, cough, pyrexia, and oropharyngeal pain. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Sobi, Inc. at 1-866-773-5274 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trial 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 to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

Pregnancy: May cause fetal harm ( 8.1 ) Lactation: Advise not to breastfeed. ( 8.2 ) 8.1 Pregnancy Risk Summary Based on findings from animal reproduction studies, DOPTELET may cause fetal harm when administered to a pregnant woman ( see Data ). The available data on DOPTELET in pregnant women are insufficient to inform a drug-associated risk of adverse developmental outcomes. In animal reproduction studies, oral administration of avatrombopag resulted in adverse developmental outcomes when administered during organogenesis in rabbits and during organogenesis and the lactation period in rats. However, these findings were observed at exposures based on an AUC substantially higher than the AUC observed in patients at the maximum recommended dose of 60 mg once daily. Advise pregnant women of the potential risk to a fetus. 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 2-4% and 15-20%, respectively.

Metabolism

The pharmacokinetics of avatrombopag were determined following administration of DOPTELET tablets, and pharmacokinetic parameters are presented as geometric mean (CV%) unless otherwise specified. Avatrombopag demonstrated dose-proportional pharmacokinetics after single doses from 10 mg (0.5 times the lowest approved dosage) to 80 mg (1.3 times the highest recommended dosage).

In the event of overdose, platelet count may increase excessively and result in thrombotic or thromboembolic complications. Closely monitor the patient and platelet count. Treat thrombotic complications in accordance with standard of care. No antidote for DOPTELET overdose is known. Hemodialysis is not expected to enhance the elimination of DOPTELET because avatrombopag is only approximately 6% renally excreted and is highly bound to plasma proteins. Consider contacting the Poison Help line (1-800-222-1222) or a medical toxicologist for additional overdose management recommendations.

• May 21, 2018NDANDA210238Akarx Inc
• Jul 24, 2025NDANDA219696Akarx Inc

1. 1Platelet Count Decreased36716%
2. 2Drug Ineffective32114%
3. 3Headache2159.1%
4. 4Platelet Count Increased1928.2%
5. 5Off Label Use1626.9%
6. 6Fatigue1606.8%
7. 7Platelet Count Abnormal1114.7%
8. 8Nausea1054.5%
9. 9Product Dose Omission Issue964.1%
10. 10Death954.0%
11. 11Diarrhoea823.5%
12. 12Inappropriate Schedule Of Product Administration793.4%
13. 13Arthralgia682.9%
14. 14Dizziness612.6%
15. 15Asthenia592.5%

The 10 most recently updated of 62 ClinicalTrials.gov registrations naming Avatrombopag 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
• Evaluation of Avatrombopag for the Treatment of Thrombocytopenia in Japanese Adults With Chronic ITP
  Completed · Phase 3 · Interventional · 19 enrolled · Sobi, Inc.
  NCT05369208updated 2026-06-03
• Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months
  Completed · Phase 3 · Interventional · 75 enrolled · Sobi, Inc.
  NCT04516967updated 2026-06-03
• Avatrombopag for Severe Thrombocytopenia After Liver Transplantation: A Retrospective Cohort Study
  Completed · Observational · 134 enrolled · Shanghai Zhongshan Hospital
  NCT07600905updated 2026-05-22
• Avatrombopag vs. Placebo for CIT in GI Malignancies
  Active not recruiting · Phase 2 · Interventional · 60 enrolled · Hanny Al-Samkari, MD
  NCT05772546updated 2026-03-13
• Avatrombopag Combined With All-trans Retinoic Acid in the Treatment of Primary Immune Thrombocytopenia
  Not yet recruiting · Phase 2 · Phase 3 · Interventional · 248 enrolled · Institute of Hematology & Blood Diseases Hospital, China
  NCT07278908updated 2025-12-12
• The Holistic Study
  Not yet recruiting · Phase 3 · Interventional · 220 enrolled · Ostfold Hospital Trust
  NCT07051915updated 2025-09-09
• Exploring the Efficacy, Safety of a Modified Starting Dosage of Avatrombopag in Immune Throbocytopenia (ITP) - a Pilot Study
  Recruiting · Phase 4 · Interventional · 25 enrolled · Al-Mustansiriyah University
  NCT07133659updated 2025-08-21
• An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® in Patients With ITP
  Completed · Observational · 199 enrolled · Swedish Orphan Biovitrum
  NCT04943042updated 2025-07-14
• CsA+ATG+AVA vs. CsA+AVA for the Treatment of Newly-diagnosed SAA in the Elderly
  Completed · Phase 4 · Interventional · 86 enrolled · Peking Union Medical College Hospital
  NCT05996393updated 2025-04-30

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

• CYP2C9CPIC B/C (provisional)FDA label: Informative PGx

Frequently asked questions

How does Avatrombopag work?

Avatrombopag is an orally bioavailable, small molecule TPO receptor agonist that stimulates proliferation and differentiation of megakaryocytes from bone marrow progenitor cells, resulting in an increased production of platelets. Avatrombopag does not compete with TPO for binding to the TPO receptor and has an additive effect with TPO on platelet production.

What is Avatrombopag used for?

According to FDA labeling, Avatrombopag carries indications including: DOPTELET is a thrombopoietin receptor agonist indicated for the treatment of: Thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure. ( 1.1 ) Thrombocytopenia in adult patients with chronic immune thrombocytopenia who have had an insufficient response to a previous treatment.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Avatrombopag?

Avatrombopag is classified as Other systemic hemostatics, ATP-Binding Cassette (ABC) Transporter Interactions, Thrombopoietin Receptor Agonists, Increased Megakaryocte Cell Production, Increased Platelet Production.

What are the brand names for Avatrombopag?

Avatrombopag is marketed under brand names including Doptelet.

What are the contraindications for Avatrombopag?

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