OncoSignal
Live from Open Targets Platform, fetched 2026-11-11 15:38 UTC. Every row links to its source record.

ACVR1 activin A receptor type 1

ENSG00000115170 · protein coding · Open Targets profile

Bone morphogenetic protein (BMP) type I receptor that is involved in a wide variety of biological processes, including bone, heart, cartilage, nervous, and reproductive system development and regulation (PubMed:20628059, PubMed:22977237). As a type I receptor, forms heterotetrameric receptor complexes with the type II receptors AMHR2, ACVR2A or ACVR2B (PubMed:17911401). Upon binding of ligands such as BMP7 or GDF2/BMP9 to the heteromeric complexes, type II receptors transphosphorylate ACVR1 intracellular domain (PubMed:25354296). In turn, ACVR1 kinase domain is activated and subsequently phosphorylates SMAD1/5/8 proteins that transduce the signal (PubMed:9748228). In addition to its role in mediating BMP pathway-specific signaling, suppresses TGFbeta/activin pathway signaling by interfering with the binding of activin to its type II receptor (PubMed:17911401). Besides canonical SMAD signaling, can activate non-canonical pathways such as p38 mitogen-activated protein kinases/MAPKs (By similarity). May promote the expression of HAMP, potentially via its interaction with BMP6 (By similarity). {ECO:0000250|UniProtKB:P15261, ECO:0000250|UniProtKB:P37172, ECO:0000269|PubMed:17911401, ECO:0000269|PubMed:20628059, ECO:0000269|PubMed:22977237, ECO:0000269|PubMed:25354296, ECO:0000269|PubMed:9748228}.

5 of the 6 drugs recorded against ACVR1 are approved

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.

4 small molecule drugs at Approved: PACRITINIB, MOMELOTINIB DIHYDROCHLORIDE MONOHYDRATE, PACRITINIB CITRATE, MOMELOTINIB1 protein drug at Approved: DIBOTERMIN ALFA1 protein drug at Withdrawn: EPTOTERMIN ALFASmall molecule 4Protein 2Approved 5Withdrawn 1

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 Withdrawn means the drug was pulled from the market or from development.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.

In 11 of 15 cancers, the evidence for ACVR1 comes mainly from mutations found in tumours

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.

Inherited (germline) variantsMutations in tumours (somatic)
00.250.50.751myelofibrosismyelofibrosisprimary myelofibrosisprimary myelofibrosispancreatic ductal adenocarcinomapancreatic ductal adenocarcinomapancreatic ductal adenocarcinoma: somatic 0.61myeloproliferative disordermyeloproliferative disorderacquired polycythemia veraacquired polycythemia veraendometrial cancerendometrial cancerendometrial cancer: somatic 0.61malignant gliomamalignant gliomamalignant glioma: somatic 0.61kidney neoplasmkidney neoplasmkidney neoplasm: somatic 0.61carcinoma of liver and intrahepatic bil…carcinoma of liver and intrahepatic biliary tractcarcinoma of liver and intrahepatic biliary tract: somatic 0.61lymphoid neoplasmlymphoid neoplasmlymphoid neoplasm: somatic 0.61bile duct carcinomabile duct carcinomabile duct carcinoma: somatic 0.61breast ductal adenocarcinomabreast ductal adenocarcinomabreast ductal adenocarcinoma: somatic 0.61HER2 positive breast carcinomaHER2 positive breast carcinomaHER2 positive breast carcinoma: somatic 0.61esophageal adenocarcinomaesophageal adenocarcinomaesophageal adenocarcinoma: somatic 0.61ovarian endometrioid adenocarcinoma wit…ovarian endometrioid adenocarcinoma with squamous differentiationovarian endometrioid adenocarcinoma with squamous differentiation: somatic 0.61

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
Structure with Ligand, High-Quality Ligand, High-Quality Pocket, Druggable Family
Antibody
GO CC high conf, UniProt SigP or TMHMM
PROTAC
Database Ubiquitination, Half-life Data, Small Molecule Binder
Other modalities
Approved Drug

gnomAD loss of function constraint: o/e 0.6656500101089478 (upper bound 0.8724899888038635). 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 55 registry trials, updated in the last three years, of the most advanced drugs against ACVR1 (pacritinib, momelotinib dihydrochloride monohydrate, dibotermin alfa, momelotinib), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.

29 of 49 trials of drugs against ACVR1 have sites only in United States

Registry trials of pacritinib, momelotinib dihydrochloride monohydrate, dibotermin alfa, momelotinib 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; 38 of 49 have all sites in one country.

Sites only in this groupSites here and elsewhere
United StatesUnited States: 29 trials with sites only hereUnited States: 11 trials with sites here and elsewhere40ChinaChina: 0 trials with sites only hereChina: 2 trials with sites here and elsewhere2EuropeEurope: 3 trials with sites only hereEurope: 9 trials with sites here and elsewhere12JapanJapan: 0 trials with sites only hereJapan: 4 trials with sites here and elsewhere4OtherOther: 6 trials with sites only hereOther: 9 trials with sites here and elsewhere15

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. 6 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.

32 trials of drugs against ACVR1 were stopped early; 13 give no reason

Terminated, withdrawn and suspended interventional trials of pacritinib, momelotinib dihydrochloride monohydrate, dibotermin alfa, momelotinib, 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 32.

Paediatric incentives and obligations for ACVR1's drugs

For pacritinib, momelotinib dihydrochloride monohydrate, dibotermin alfa, momelotinib: 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: yes · ACVR1 is on the list. Membership alone neither requires nor waives a pediatric study.
    Rule: 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-001656-PIP01-14 (Momelotinib, Treatment of acute lymphoblastic leukaemia;Treatment of essential thrombocythaemia;Treatment of polycythaemia vera;Treatment of post-essential thrombocythaemia myelofibrosis;Treatment of post-polycythaemia vera myelofibrosis): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 10 Jul 2015; EMEA-001656-PIP02-19 (Momelotinib, Treatment of myelofibrosis): W: decision granting a waiver in all age groups for all conditions or indications, 17 Jun 2019; EMEA-001621-PIP01-14 (Pacritinib, Treatment of post-essential thrombocythaemia myelofibrosis;Treatment of post-polycythaemia vera myelofibrosis): W: decision granting a waiver in all age groups for all conditions or indications, 6 Aug 2014
    Rule: 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/3/26/3223 positive 20 Apr 2026: Treatment of VEXAS syndrome; EU/3/11/888 positive 5 Aug 2011: Treatment of primary myelofibrosis; EU/3/11/886 positive 5 Aug 2011: Treatment of post-polycythaemia vera myelofibrosis; EU/3/11/887 positive 5 Aug 2011: Treatment of post-essential thrombocythaemia myelofibrosis
    Rule: 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 data download (dated snapshot), 2026-11-11
  • Authorised in the EU: yes · Omjjara: authorised since 25 Jan 2024, orphan medicine; Inductos: authorised since 9 Sept 2002
    Rule: 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 link
    Rule: 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 pacritinib, momelotinib dihydrochloride monohydrate, dibotermin alfa, momelotinib
    Rule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-11
  • Registered studies open to children: yes · 7 interventional studies of pacritinib, momelotinib dihydrochloride monohydrate, dibotermin alfa, momelotinib include patients under 18; 2 are recruiting, not yet recruiting or active
    Rule: The registry's age group includes Child (birth to 17 years), which also counts studies that set no minimum age. Source: ClinicalTrials.gov search, 2026-11-11

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 6 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.

DrugTypeHighest stageIndicationsSource records
PACRITINIBSmall moleculeapprovalmyelofibrosis, neoplasm, primary myelofibrosis, marginal zone lymphoma, lymphoplasmacytic lymphoma, follicular lymphoma, B-cell chronic lymphocytic leukemia, mantle cell lymphoma, +26 moreOpen Targets · ChEMBL
MOMELOTINIB DIHYDROCHLORIDE MONOHYDRATESmall moleculeapprovalmyelofibrosisOpen Targets · ChEMBL
PACRITINIB CITRATESmall moleculeapprovalacquired polycythemia vera, primary myelofibrosisOpen Targets · ChEMBL
DIBOTERMIN ALFAProteinphase 4spinal fusion, tibia fracture, bone disorder, osteoporosisOpen Targets · ChEMBL
MOMELOTINIBSmall moleculephase 4myelofibrosis, Splenomegaly, myeloproliferative disorder, neoplasm, primary myelofibrosis, pancreatic ductal adenocarcinoma, VEXAS syndrome, acquired polycythemia vera, +8 moreOpen Targets · ChEMBL
EPTOTERMIN ALFAProteinwithdrawaltibia fracture, spondylolisthesis, bone disorder, osteoarthritis, osteoarthritis, kneeOpen Targets · ChEMBL