Drugs / Avatrombopag
last change Jul 2026 re-read 2 minutes ago

Avatrombopag

Small molecule targets MPL via activation

Developed for
thrombocytopenia · aplastic anemia · autoimmune thrombocytopenia · autoimmune thrombocytopenic purpura · cirrhosis of liver · connective tissue disorder · Evans syndrome · liver disorder
+5 more · platelet disorder, undefined · liver failure · thrombocytopenia due to immune destruction · thrombocytopenic purpura · primary thrombocytopenia
Investigated by
Eisai Inc. · Institute of Hematology & Blood Diseases Hospital, China · Sobi, Inc. · Peking Union Medical College Hospital · First Affiliated Hospital of Harbin Medical University · Henan Cancer Hospital
+48 more · Peking University People's Hospital · The First Affiliated Hospital of Nanchang University · The Second Affiliated Hospital of Kunming Medical University · Tianjin Medical University Second Hospital · 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China · Al-Mustansiriyah University · Anhui Provincial Cancer Hospital · Anhui Provincial Hospital · Ayman Saad · Chinese PLA General Hospital · First Affiliated Hospital of Xinjiang Medical University · Fosun Pharmaceutical Distribution Jiangsu co., Limited · Hanny Al-Samkari, MD · Haukeland University Hospital · Helse Fonna · Helse Nord-Trøndelag HF · Helse Stavanger HF · Hvidovre University Hospital · Memorial Sloan Kettering Cancer Center · Nordland Hospital · Oslo University Hospital · Ostfold Hospital Trust · Second Affiliated Hospital of Guangzhou Medical University · Second Hospital of Shanxi Medical University · Shanxi Bethune Hospital · Sichuan Provincial People's Hospital · Sorlandet Hospital HF · St. Olavs Hospital · Sun Yat-sen University · Swedish Orphan Biovitrum · Sykehuset i Vestfold HF · Taihe Hospital, Hubei University of Medicine · Tang-Du Hospital · The First Affiliated Hospital of Soochow University · The First Affiliated Hospital of Zhengzhou University · The First Hospital of Hebei Medical University · The First Hospital of Jilin University · Third Affiliated Hospital, Sun Yat-Sen University · Tianjin Children's Hospital · Tianjin Medical University Cancer Institute and Hospital · Tianjin People's Hospital · Tianjin Third Central Hospital · Tongji Hospital · University Hospital of North Norway · University Hospital, Akershus · University of Rochester · Xiangya Hospital of Central South University · Xiyuan Hospital of China Academy of Chinese Medical Sciences

Regulatory milestones approvals, filings & regulatory actions · 4 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Label expansions (2) US (FDA) DOPTELET 2019-06-26 – 2025-07-24 fda.gov
Approved EU (EMA) Doptelet 2019-06-20 europa.eu
Approved US (FDA) DOPTELET 2018-05-21 fda.gov

Trials 53 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 01 trial
Phase 0 NCT04931849 May 2022 → Oct 2023 thrombocytopenia University of Rochester Withdrawn No outcome recorded
Phase 110 trials
Phase 1 NCT02809768 Apr → Aug 2016 Eisai Inc. Completed No outcome recorded
Phase 1 NCT02039076 Dec 2013 → Jun 2014 thrombocytopenia Eisai Inc. Completed No outcome recorded
Phase 1 NCT01774773 Jan → Jul 2013 Eisai Inc. Completed No outcome recorded
Phase 1 NCT01759394 Oct 2012 → Jan 2013 Eisai Inc. Completed No outcome recorded
Phase 1 NCT01549054 Jan → Jun 2012 autoimmune thrombocytopenic purpura Eisai Inc. Completed No outcome recorded
Phase 1 NCT01437384 Sep 2011 → Mar 2012 autoimmune thrombocytopenic purpura Eisai Inc. Completed No outcome recorded
Phase 1 NCT01327872 Feb → Apr 2011 autoimmune thrombocytopenic purpura Eisai Inc. Completed No outcome recorded
Phase 1 NCT01289509 Dec 2010 → Mar 2011 autoimmune thrombocytopenic purpura Eisai Inc. Completed No outcome recorded
Phase 1 NCT01251731 Jul 2010 → Jan 2011 Eisai Inc. Completed No outcome recorded
Phase 1 NCT01260155 Jan → Jun 2010 Eisai Inc. Completed No outcome recorded
Phase 218 trials
Phase 2 NCT06281327 Mar 2024 → Dec 2025 overdue autoimmune thrombocytopenia Institute of Hematology & Blood Diseases Hospital, China Recruiting No outcome recorded
Phase 2 NCT06254287 Feb → Dec 2024 aplastic anemia Institute of Hematology & Blood Diseases Hospital, China Unknown No outcome recorded
Phase 2 NCT05772546 Nov 2023 → Jan 2026 overdue digestive system cancer, thrombocytopenia Hanny Al-Samkari, MD Active not recruiting No outcome recorded
Phase 2 NCT06004752 Aug 2023 → Aug 2024 aplastic anemia Peking Union Medical College Hospital Unknown No outcome recorded
Phase 2 NCT06001567 Aug 2023 → Aug 2024 hepatocellular carcinoma, thrombocytopenia Second Affiliated Hospital of Guangzhou Medical University Unknown No outcome recorded
Phase 2 NCT04312789 Dec 2022 platelet disorder, undefined Ayman Saad Withdrawn No outcome recorded
Phase 2 NCT05720234 Nov 2022 → Nov 2023 aplastic anemia Institute of Hematology & Blood Diseases Hospital, China Unknown No outcome recorded
Phase 2 NCT05218226 Feb 2022 → Dec 2024 thrombocytopenia Institute of Hematology & Blood Diseases Hospital, China Unknown No outcome recorded
Phase 2 NCT05143892 Dec 2021 → Dec 2022 platelet disorder, undefined The First Affiliated Hospital of Soochow University Unknown No outcome recorded
Phase 2 NCT04993885 Sep 2021 → Jul 2024 overdue Evans syndrome, connective tissue disorder Institute of Hematology & Blood Diseases Hospital, China Recruiting No outcome recorded
Phase 2/34 trials
Phase 2/3 NCT07278908 Dec 2025 → Dec 2028 expected autoimmune thrombocytopenia Institute of Hematology & Blood Diseases Hospital, China Not yet recruiting No outcome recorded
Phase 2/3 NCT06202625 May → Oct 2024 overdue disease related to hematopoietic stem cell transplant, thrombocytopenia Peking University People's Hospital Recruiting No outcome recorded
Phase 2/3 NCT04915482 Jun 2021 → Jan 2025 Evans syndrome, connective tissue disorder Institute of Hematology & Blood Diseases Hospital, China Unknown No outcome recorded
Phase 2/3 NCT04728789 Feb 2021 → Dec 2022 aplastic anemia Peking Union Medical College Hospital Unknown No outcome recorded
Phase 310 trials
Phase 3 NCT07051915 Sep 2025 → Jan 2028 expected thrombocytopenic purpura Ostfold Hospital Trust Not yet recruiting No outcome recorded
Phase 3 NCT05369208 Jun 2022 → Jan 2024 autoimmune thrombocytopenia Sobi, Inc. Completed No outcome recorded
Phase 3 NCT05046327 Oct 2021 → Apr 2024 autoimmune thrombocytopenia Sobi, Inc. Withdrawn No outcome recorded
Phase 3 NCT04516967 Mar 2021 → Nov 2023 autoimmune thrombocytopenia Sobi, Inc. Completed No outcome recorded
Phase 3 NCT03471078 Oct 2018 → Aug 2020 thrombocytopenia Sobi, Inc. Completed No outcome recorded
Phase 3 NCT03326843 Mar 2018 → Feb 2019 thrombocytopenia Sobi, Inc. Terminated No outcome recorded
Phase 3 NCT01972529 Feb 2014 → Jan 2017 thrombocytopenia Eisai Inc. Completed No outcome recorded
Phase 3 NCT01976104 Nov 2013 → Jan 2017 thrombocytopenia Eisai Inc. Completed No outcome recorded
Phase 3 NCT01433978 Mar 2012 → Sep 2013 autoimmune thrombocytopenic purpura Eisai Inc. Terminated No outcome recorded
Phase 3 NCT01438840 Feb 2012 → Nov 2013 thrombocytopenia due to immune destruction Eisai Inc. Completed No outcome recorded
Phase 46 trials
Phase 4 NCT07133659 Jun 2025 → Jul 2026 overdue autoimmune thrombocytopenia Al-Mustansiriyah University Recruiting No outcome recorded
Phase 4 NCT05382013 Apr 2022 → Feb 2024 chronic hepatitis B virus infection, thrombocytopenia Third Affiliated Hospital, Sun Yat-Sen University Unknown No outcome recorded
Phase 4 NCT04915287 Jun 2021 → Jun 2023 liver disorder, thrombocytopenia Chinese PLA General Hospital Unknown No outcome recorded
Phase 4 NCT04845659 Jun 2021 → Jun 2022 cirrhosis of liver, thrombocytopenia Hvidovre University Hospital Unknown No outcome recorded
Phase 4 NCT04638829 Mar 2021 → Jan 2024 autoimmune thrombocytopenia Sobi, Inc. Completed No outcome recorded
Phase 4 NCT04906083 Feb 2021 → Dec 2022 cirrhosis of liver, liver failure, thrombocytopenia Tongji Hospital Unknown No outcome recorded
Phase not applicable4 trials
NCT05571332 Jun 2022 → Dec 2023 aplastic anemia Institute of Hematology & Blood Diseases Hospital, China Unknown No outcome recorded
NCT05518331 Jun 2022 → Jun 2025 aplastic anemia Institute of Hematology & Blood Diseases Hospital, China Unknown No outcome recorded
NCT05433922 Jun 2022 → Dec 2023 aplastic anemia Institute of Hematology & Blood Diseases Hospital, China Unknown No outcome recorded
NCT04609891 May 2020 → May 2021 thrombocytopenia Anhui Provincial Cancer Hospital Completed No outcome recorded
Also used as a comparator or background therapy in 4 trials
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 21 trial
Phase 2 NCT07421167 Background Jun 2026 → Apr 2028 expected Evans syndrome Novartis Pharmaceuticals Recruiting No outcome recorded
Phase 31 trial
Phase 3 NCT05907499 Background Jul 2023 → Jul 2026 overdue The First Affiliated Hospital of Soochow University Not yet recruiting No outcome recorded
Phase 42 trials
Phase 4 NCT05996393 Background Sep 2023 → Feb 2025 aplastic anemia Peking Union Medical College Hospital Completed No outcome recorded
Phase 4 NCT05718856 Comparator Feb 2023 → May 2025 primary thrombocytopenia Institute of Hematology & Blood Diseases Hospital, China Unknown No outcome recorded

Evidence & citations 13 cited values

Every value below carries the sentence it was read from. 60 sources stand behind the page.

FieldValueCited text
Known as Avatrombopag “Eltrombopag initial dose: 25 mg orally once a day, may increase to up to 75 mg/day, when platelet count ≤ 30 × 109/L; Avatrombopag initial dose: 20 mg orally once a day, may...” NCT05907499
48

“Three TPO-RAs could be used in this study, including eltrombopag, hetrombopag and avatrombopag.” NCT05718856

“The starting dose of avatrombopag is 20-40mg once daily.” NCT04915482

avatrombopagNCT07421167

NCT01355289

NCT04312789

NCT06281327

NCT05720234

NCT01433978

NCT05369208

NCT06254287

NCT05571332

NCT01976104

NCT00625443

NCT06001567

NCT03326843

NCT04797182

NCT07133659

NCT05772546

NCT07278908

NCT05996393

NCT05218226

NCT04437953

NCT05518331

NCT04993885

NCT04906083

NCT04516967

NCT02809768

NCT05433922

NCT01759394

NCT05143892

NCT00914927

NCT07051915

NCT01972529

NCT04915287

NCT01438840

NCT04638829

NCT04728789

NCT04931849

NCT00441090

NCT02227693

NCT02039076

NCT06202625

NCT03471078

NCT06004752

NCT04896528

NCT05382013

NCT05046327

NCT04609891

Known as Akr-501 ChEMBL registry synonym — accepted as the source's own label CHEMBL2103883
Known as AVA Avatrombopag (AVA) is prescribed for thrombocytopenic diseases but not for NSAA.” PMID 37318085 Dec 2023
1

“addition of avatrombopag (AVA), which has a slight adverse reaction” NCT06004752

Known as Avatrombopag maleate Avatrombopag maleate 40 mg (2 x 20 mg tablets- all doses are expressed as avatrombopag, the amount of free base)” NCT01759394
Known as Doptelet ClinicalTrials.gov intervention name — accepted as the source's own label NCT04845659
Known as E-5501 MALEATE ChEMBL registry synonym — accepted as the source's own label CHEMBL2103883
Known as E5501 E5501 (avatrombopag) 20-mg tablets” NCT02227693
7

NCT01774773

NCT01549054

NCT01289509

NCT01260155

NCT01327872

NCT01437384

NCT01251731

Known as YM-301477 ChEMBL registry synonym — accepted as the source's own label CHEMBL2103883
Known as YM-477 ChEMBL registry synonym — accepted as the source's own label CHEMBL2103883
Action Activate “avatrombopag, an investigational thrombopoietin-receptor agonist” PMID 24802775 May 2014
1

“Thrombopoietin receptor agonist” CHEMBL2103883

Modality Small molecule “once-daily oral avatrombopag (2.5, 5, 10, or 20 mg), an investigational nonpeptide TPO-RA active in humans” PMID 24802775 May 2014
Route Oral “once-daily oral avatrombopag” PMID 24802775 May 2014
Target MPL “Thrombopoietin receptor agonist” CHEMBL2103883