MPL MPL proto-oncogene, thrombopoietin receptor
ENSG00000117400 · protein coding · Open Targets profile
Receptor for thrombopoietin that regulates hematopoietic stem cell renewal, megakaryocyte differentiation, and platelet formation. Upon activation by THPO, induces rapid tyrosine phosphorylation and activation of JAK2, providing docking sites for many signaling proteins such as STAT5, SHIP/INPP5D, GRB2, SOS1 and PI3K (PubMed:15899890, PubMed:37633268). In turn, These signaling cascades lead to the proliferation, survival, and differentiation of megakaryocytes, ultimately leading to increased platelet production. .
Each band is a group of drugs. It runs from the kind of drug on the left to the furthest stage that drug has reached in any disease on the right. Thicker bands mean more drugs.
Approved bands are highlighted. Withdrawn means the drug was pulled from the market or from development. A combined phase counts as the later one (Phase 1/2 is shown as Phase 2). Hover a band for the drug names. Source: Open Targets Platform, fetched live.
Each row is a cancer, strongest overall evidence first. The two dots show how strong the inherited (germline) and tumour-mutation (somatic) evidence is; the line between them is the gap.
Scores run from 0 (no evidence) to 1 (strongest). A missing dot at 0 means Open Targets holds no evidence of that kind. Source: Open Targets Platform, fetched live.
Tractability
- Small molecule
- Approved Drug, High-Quality Ligand, Druggable Family
- Antibody
- UniProt loc high conf, GO CC high conf, UniProt SigP or TMHMM, Human Protein Atlas loc
- PROTAC
- UniProt Ubiquitination, Database Ubiquitination, Small Molecule Binder
- Other modalities
- Approved Drug
gnomAD loss of function constraint: o/e 0.8470799922943115 (upper bound 1.0291999578475952). Low values mean loss of function is poorly tolerated, a safety flag for inhibition.
Who runs trials of these drugs
Principal investigators and study chairs on 145 registry trials, updated in the last three years, of the most advanced drugs against MPL (eltrombopag choline, avatrombopag, eltrombopag olamine, lusutrombopag, eltrombopag, romiplostim, rafutrombopag, hetrombopag olamine), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Lei ZhangThrombosis and Haemostasis Diagnosis Treatment Center · 9 of 9 trials Phase 2+
- Xiaohui ZhangPeking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Collaborative Innovation Center of Hematology · 4 of 4 trials Phase 2+Ph2NCT07586813 · Romiplostim N01 Plus ATRA for Persistent Isolated Chemotherapy-Induced Thrombocytopenia After Complete Remission of Gastrointestinal Solid Tumorsbrief
- Neal S YoungNational Heart, Lung, and Blood Institute (NHLBI) · 2 of 2 trials Phase 2+
- Oksana A. MarkovaAO GENERIUM · 2 of 2 trials Phase 2+
- Tienan ZhuPeking Union Medical College Hospital · 2 of 2 trials Phase 2+
- Uday PopatM.D. Anderson Cancer Center · 2 of 2 trials Phase 2+
- Heng MeiUnion Hospital, Tongji Medical College, Huazhong University of Science and Technology · 1 of 2 trials Phase 2+
- Alexandra SchifferliUniversity Children's Hospital Basel · 1 of 1 trials Phase 2+
- Amanda GrimesBaylor College of Medicine · 1 of 1 trials Phase 2+
- Andre LarochelleNational Heart, Lung, and Blood Institute (NHLBI) · 1 of 1 trials Phase 2+
- Antonio RisitanoFederico II Medical School, Haematology Division, Napels · 1 of 1 trials Phase 2+
- Arnaud PIGNEUXFrench Innovative Leukemia Organization (FILO) · 1 of 1 trials Phase 2+
- Bhavisha A PatelNational Heart, Lung, and Blood Institute (NHLBI) · 1 of 1 trials Phase 2+
- Bing BingPeking Union Medical College Hospital · 1 of 1 trials Phase 2+
- Bing HanPeking Union Medical College · 1 of 1 trials Phase 2+
Registry trials of eltrombopag choline, avatrombopag, eltrombopag olamine, lusutrombopag, eltrombopag, romiplostim, rafutrombopag, hetrombopag olamine updated in the last three years, counted by the country group of their listed sites. A trial with sites in several groups counts in each; 109 of 122 have all sites in one country.
Site counts are not patients per country, and trials registered only outside ClinicalTrials.gov are not covered. Where sites are is a fact about the record, not a judgement of its quality. Europe is the EU and EEA, the UK and Switzerland. 23 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
42 trials of drugs against MPL were stopped early; 2 give no reason
Terminated, withdrawn and suspended interventional trials of eltrombopag choline, avatrombopag, eltrombopag olamine, lusutrombopag, eltrombopag, romiplostim, rafutrombopag, hetrombopag olamine, newest update first, live from ClinicalTrials.gov. Each reason is quoted exactly as the registry gives it; we do not sort reasons into safety, efficacy or business. Dates are the last registry update. Showing the 25 most recent of 42.
- withdrawn · 2026-10-16 · Anjali Sharathkumar · Romiplostim“Withdrawn in HawkIRB”
- terminated · 2026-10-09 · University of California, Davis · Eltrombopag“PI discretion; suspended while CAPA is reviewed”
- suspended · 2026-10-09 · Abhay Singh, MD MPH · Eltrombopag (EPAG)“Funding unavailable”
- withdrawn · 2026-06-04 · Peking Union Medical College Hospital · Romiplostim N01“The registration of this research plan was incorrect. A new registration has been done.”
- withdrawn · 2026-06-04 · Peking Union Medical College Hospital · Romiplostim N01“The registration of this research plan was incorrect. A new registration has been re-applied for.”
- terminated · 2025-09-30 · University Hospital, Lille · eltrombopag“inclusions difficulties.”
- terminated · 2025-05-28 · Memorial Sloan Kettering Cancer Center · Romiplostim“Lack of accrual”
- withdrawn · 2025-01-15 · University of Rochester · Avatrombopag“PI and Sponsor decided to not proceed with the trial.”
- terminated · 2024-03-29 · Technische Universität Dresden · Eltrombopag“Due to COVID-19 pandemia, recruitment was insufficient to reach required sample size in an acceptable time line. Furthermore, new drugs have recently been licensed resulting in alternative treatment regimens with a better prognosis.”
- terminated · 2024-02-29 · Novartis Pharmaceuticals · Eltrombopag“In the context of COVID-19 pandemic, the benefit / risk ratio of the participation of a patient with pancytopenia could be compromised. The decision of this premature termination was not the consequence of any safety reason inherent to the drug.”
- withdrawn · 2023-09-29 · Amgen · Romiplostim“Operational”
- withdrawn · 2023-09-28 · Jiangsu HengRui Medicine Co., Ltd. · matched by the registry's drug synonyms“Sponsor R \& D Strategy Adjustment”
- suspended · 2023-09-15 · University of California, Davis · matched by the registry's drug synonyms“low enrollment”
- withdrawn · 2023-05-24 · European Organisation for Research and Treatment of Cancer - EORTC · Romiplostim“Amgen withdrew interest in providing further financial support”
- terminated · 2022-11-07 · H. Lee Moffitt Cancer Center and Research Institute · Eltrombopag“Lack of efficacy”
- terminated · 2022-07-22 · Roswell Park Cancer Institute · eltrombopag olamine“Sponsor wanted study rewritten”
- withdrawn · 2021-12-09 · Ayman Saad · Avatrombopag“Drug availability”
- withdrawn · 2021-10-22 · Sobi, Inc. · Avatrombopag 20 mg Oral Tablet“Corporate Decision”
- terminated · 2021-08-05 · Abramson Cancer Center at Penn Medicine · EltrombopagNo reason given
- terminated · 2021-03-18 · Shionogi · Lusutrombopag“This study was terminated before the planned sample size was enrolled because results indicated that a higher dose was necessary to elicit an efficacy effect.”
- terminated · 2021-02-26 · Shionogi · Lusutrombopag“The study was terminated due to cessation of clinical development of S-888711 for chronic ITP based on a business decision by the Sponsor.”
- terminated · 2021-02-08 · Gesellschaft fur Medizinische Innovation - Hamatologie und Onkologie mbH · Romiplostim“due to lack of recruitment”
- withdrawn · 2020-12-08 · Memorial Sloan Kettering Cancer Center · Avatrombopag“Lack of accrual”
- terminated · 2020-11-03 · PrECOG, LLC. · Phase I- Cytarabine & Eltrombopag“Study stopped per Novartis request due to futility from another study.”
- terminated · 2020-04-15 · Sobi, Inc. · Avatrombopag 60 mg“Study was terminated due to enrollment challenges.”
Paediatric incentives and obligations for MPL's drugs
For eltrombopag choline, avatrombopag, eltrombopag olamine, lusutrombopag, eltrombopag, romiplostim, rafutrombopag, hetrombopag olamine: what EMA and FDA publish about paediatric plans, orphan status and pediatric approvals, and how many registered studies take children. Each line is a yes, no or not published fact from the record linked under it; none of it is a forecast.
- FDA pediatric target list: no · MPL is not on the listRule: Target is on FDA's Pediatric Molecular Target List (relevant targets). Source: FDA Pediatric Molecular Target List (Listed as February 2026 on the FDA Pediatric Oncology page; the file served is 9.7.22_the_relevant_molecular_target_list.xlsx, whose Summary of Changes sheet is headed 'SEPTEMBER 2022 UPDATE' (footnote date 2022-09-01)), 2026-11-11
- EU paediatric investigation plan: yes · EMEA-001136-PIP01-11-M03 (avatrombopag maleate, Treatment of Idiopathic Thrombocytopenia Purpura (ITP);Treatment of Thrombocytopenic Purpura Secondary to Liver Disease): PM: decision on the application for modification of an agreed PIP, 6 May 2024; compliance positive 26 Jul 2024; EMEA-001136-PIP02-19-M01 (avatrombopag, Treatment of chemotherapy-induced thrombocytopenia): PM: decision on the application for modification of an agreed PIP, 9 Dec 2020; EMEA-000170-PIP03-13-M04 (eltrombopag, Treatment of aplastic anaemia): PM: decision on the application for modification of an agreed PIP, 22 Dec 2020; EMEA-000653-PIP01-09-M05 (romiplostim, Treatment of disease-related thrombocytopenia in myelodysplastic syndrome;Treatment of idiopathic thrombocytopenic purpura as a model for immunomodulation): PM: decision on the application for modification of an agreed PIP, 11 Aug 2017; compliance positive 27 Mar 2020; +3 moreRule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA data download (dated snapshot), 2026-11-11
- EU orphan designation: yes · EU/3/07/467 withdrawn 3 Aug 2007: Treatment of idiopathic thrombocytopenic purpuraRule: An EU orphan designation lists the drug as its active substance (exact name) or belongs to a medicine that does; withdrawn and expired designations are listed with their status. Source: EMA EU/3/07/467, 2026-11-11
- Authorised in the EU: yes · Eltrombopag Accord: authorised since 28 Mar 2025; Mulpleo (previously Lusutrombopag Shionogi): withdrawn since 18 Feb 2019; Doptelet: authorised since 20 Jun 2019; Eltrombopag Viatris: authorised since 12 Dec 2024; +2 moreRule: An EMA medicine record lists the drug as its active substance or INN, exact name; national-only authorisations are not in this file. Source: EMA data download (dated snapshot), 2026-11-11
- FDA orphan designation: not published · FDA's orphan designation database turns away automated requests, so this app does not read it; search it by hand at the linkRule: Would be: an FDA orphan designation names the drug. Source: FDA orphan designations search
- FDA pediatric oncology approval: no · FDA's table lists no pediatric approval of eltrombopag choline, avatrombopag, eltrombopag olamine, lusutrombopag, eltrombopag, romiplostim, rafutrombopag, hetrombopag olamineRule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-12
- Registered studies open to children: yes · 64 interventional studies of eltrombopag choline, avatrombopag, eltrombopag olamine, lusutrombopag, eltrombopag, romiplostim, rafutrombopag, hetrombopag olamine include patients under 18; 16 are recruiting, not yet recruiting or activeRule: The registry's age group includes Child (birth to 17 years), which also counts studies that set no minimum age. Source: ClinicalTrials.gov search, 2026-11-12
Rare pediatric disease voucher. FDA, content current as of 29 Apr 2026: “As of February 3, 2026, enactment of the Consolidated Appropriations Act, 2026, the rare pediatric disease PRV program will sunset after September 30, 2029. FDA may not award any PRVs under this program after September 30, 2029.” Voucher sale prices are not shown here; they appear only as company-reported news. FDA: Rare Pediatric Disease Designation and Priority Review Voucher Programs
All 9 drugs and clinical candidates as a table
Each drug, stage and indication links to its Open Targets record, where the trials and approvals behind it are listed. Salt forms (for example a mesylate) are listed as Open Targets records them, as separate entries.