Drugs / Avatrombopag
Avatrombopag
also known as
E5501 · AVA · Akr-501 · Avatrombopag maleate · Doptelet · E-5501 MALEATE · YM-301477 · YM-477
Small molecule targets MPL via activation
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence 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
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| 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| Phase 0 | NCT04931849 | May 2022 → Oct 2023 | thrombocytopenia | University of Rochester | Withdrawn | No outcome recorded |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 · 2 met primary | ||||||
| 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 | digestive system cancer, thrombocytopenia | Hanny Al-Samkari, MD | Completed | 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 | NCT04896528 | Jun → Dec 2021 | thrombocytopenia | Peking Union Medical College Hospital | Unknown | No outcome recorded |
| Phase 2 | NCT04797182 | Apr 2021 → Apr 2023 | chemotherapy-induced toxicity, lymphoma, thrombocytopenia | Sun Yat-sen University | Unknown | No outcome recorded |
| Phase 2 | NCT04437953 | May → Dec 2020 | cancer, liver disorder, thrombocytopenia | Memorial Sloan Kettering Cancer Center | Withdrawn | No outcome recorded Stop: Enrollment |
| Phase 2 | NCT02227693 | Jul 2014 → Apr 2015 | thrombocytopenia | Eisai Inc. | Completed | Met primary |
| Phase 2 | NCT01355289 | Nov 2011 → Feb 2014 | thrombocytopenia | Eisai Inc. | Completed | Met primary |
| Phase 2 | NCT00914927 | May 2009 → Nov 2011 | thrombocytopenia | Eisai Inc. | Completed | No outcome recorded |
| Phase 2 | NCT00625443 | May 2007 → Jun 2009 | autoimmune thrombocytopenic purpura | Eisai Inc. | Completed | No outcome recorded |
| Phase 2 | NCT00441090 | Feb 2007 → Jan 2009 | autoimmune thrombocytopenic purpura | Eisai Inc. | Completed | 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 · 4 met primary · 1 missed | ||||||
| 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 | Met primary |
| Phase 3 | NCT03471078 | Oct 2018 → Aug 2020 | thrombocytopenia | Sobi, Inc. | Completed | Missed primary |
| Phase 3 | NCT03326843 | Mar 2018 → Feb 2019 | thrombocytopenia | Sobi, Inc. | Terminated | No outcome recorded Stop: Enrollment |
| Phase 3 | NCT01972529 | Feb 2014 → Jan 2017 | thrombocytopenia | Eisai Inc. | Completed | Met primary |
| Phase 3 | NCT01976104 | Nov 2013 → Jan 2017 | thrombocytopenia | Eisai Inc. | Completed | Met primary |
| Phase 3 | NCT01433978 | Mar 2012 → Sep 2013 | autoimmune thrombocytopenic purpura | Eisai Inc. | Terminated | No outcome recorded Stop: Enrollment |
| Phase 3 | NCT01438840 | Feb 2012 → Nov 2013 | thrombocytopenia due to immune destruction | Eisai Inc. | Completed | Met primary |
| 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 |
| — | NCT05433922 | 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 |
| — | NCT04609891 | May 2020 → May 2021 | thrombocytopenia | Anhui Provincial Cancer Hospital | Completed | No outcome recorded |
| Comparator or background therapy4 trials | ||||||
| Phase 2 | NCT07421167 Background | Aug 2026 → Jan 2029 expected | Evans syndrome | Novartis Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 3 | NCT05907499 Background | Jul 2023 → Jul 2026 overdue | — | The First Affiliated Hospital of Soochow University | Not yet recruiting | No outcome recorded |
| 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 |
News releases announcing trial results or a regulatory action · 9
| Date | Issuer | Release |
|---|---|---|
| 2024-03-25 | Sobi, Inc. | Results Sobi announces positive results from phase 3 study of Doptelet® for treatment of children and adolescents with ITP sobi.com ↗
The key secondary endpoint of two consecutive platelet counts ≥50×10 9 /L was met in 81.5% of avatrombopag subjects in comparison with 0% of placebo subjects (p= < 0.0001, 95% CI 71.1-91.8). |
| 2022-09-22 | Jemincare | Regulatory 济民可信“马来酸阿伐曲泊帕”原料药申报获CDE受理 jemincare.com ↗
旗下子公司上海济煜医药科技有限公司创新技术药物研究院承担研发的特色原料药马来酸阿伐曲泊帕已提交注册并获国家药品监督管理局药品审评中心（ CDE ）受理。 |
| 2021-01-20 | Sobi, Inc. | Regulatory Doptelet® (avatrombopag) approved in the EU for treatment of ITP sobi.com ↗
Doptelet® (avatrombopag) approved in the EU for treatment of ITP |
| 2020-12-11 | Sobi, Inc. | Regulatory Doptelet® (avatrombopag) receives positive opinion from CHMP for treatment of ITP sobi.com ↗
Today's positive opinion by the CHMP is a significant milestone for people living with ITP across Europe. |
| 2020-10-09 | Sobi, Inc. | Results Sobi announces topline phase 3 data of avatrombopag for the treatment of Chemotherapy-Induced Thrombocytopenia sobi.com ↗
Though avatrombopag increased platelet counts relative to placebo as expected, the study did not meet the composite primary endpoint of avoiding platelet transfusions, chemotherapy dose reductions by 15 per cent or greater, and chemotherapy dose delays by four days or more. |
| 2019-12-21 | Sobi, Inc. | Regulatory FDA grants avatrombopag Orphan Drug Designation for the treatment of Chemotherapy-Induced Thrombocytopenia sobi.com ↗
Dova Pharmaceuticals, Inc., a wholly owned subsidiary of Swedish Orphan Biovitrum AB (publ) (Sobi™) has been granted Orphan Drug Designation [1] (ODD) by the US Food and Drug Administration (FDA) for avatrombopag for the potential treament of Chemotherapy-Induced Thrombocytopenia (CIT). |
| 2019-12-09 | Sobi, Inc. | Results Doptelet [® ](avatrombopag) demonstrates cost-effectiveness for the treatment of thrombocytopenia in patients with chronic liver disease sobi.com ↗ |
| 2019-12-09 | Sobi, Inc. | Results Additional analyses from phase 3 study with Doptelet [® ](avatrombopag) for the treatment of chronic immune thrombocytopenia provides evidence of long-term response rates sobi.com ↗
Durable responders maintained a high rate of platelet response and complete platelet response in the extension phase, providing evidence that AVA-responding patients will likely continue to respond for extended treatment periods. |
| 2019-12-05 | Sobi, Inc. | Results Data to be highlighted at the American Society of Hematology 2019 Annual Meeting sobi.com ↗
Additional analyses from the phase 3 study - core and extension phase - with avatrombopag for the treatment of ITP confirm the long-term response rates and efficacy of treatment with avatrombopag in this indication. |
All press releases naming this drug 14 releases
Evidence & citations 13 cited values
Every value below carries the sentence it was read from. 60 sources stand behind the page.
| Field | Value | Cited 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 ↗ “avatrombopag” NCT07421167 ↗ |
| 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 20231“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 ↗ |
| 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 20141“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 ↗ |