GHSR growth hormone secretagogue receptor
ENSG00000121853 · protein coding · Open Targets profile
G protein-coupled receptor specific to ghrelin, an appetite- regulating peptide hormone commonly found in stomach (PubMed:35027551, PubMed:39833471). Upon activation, stimulates appetite and promotes growth hormone secretion (PubMed:10604470, PubMed:11322507, PubMed:35027551, PubMed:39833471). Ghrelin binding causes a conformation change that triggers signaling via guanine nucleotide- binding proteins (G proteins) and modulates the activity of downstream effectors, such as phospholipase C (By similarity). GHSR is coupled to G(q) G proteins and mediates production of diacylglycerol (DAG) and inositol 1,4,5-trisphosphate (IP3) (By similarity). Also binds other growth hormone releasing peptides (GHRP) (e.g. Met-enkephalin and GHRP- 6) as well as non-peptide, low molecular weight secretagogues (e.g. L- 692, 429, MK-0677, adenosine) (PubMed:10604470, PubMed:11322507). Also plays a role in synaptic plasticity independently of ghrelin by forming a heteromer with the dopamine receptors DRD1 and DRD2 in neurons (By similarity). Following DRD1 activation by dopamine in hippocampal neurons, GHSR acts as a coreceptor, resulting in DRD1 coupling to G(q) at the expense of G(s), initiating hippocampal synaptic plasticity involved in memory and learning (By similarity). Following DRD2 activation by dopamine in lumbosacral defecation centers, GHSR acts as a coreceptor, resulting in DRD1 coupling to G(q) at the expense of G(i)/G(o), regulating defecation (By similarity). {ECO:0000250|UniProtKB:O08725, ECO:0000250|UniProtKB:Q99P50, ECO:0000269|PubMed:10604470, ECO:0000269|PubMed:11322507, ECO:0000269|PubMed:35027551, ECO:0000269|PubMed:39833471}.
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 Withdrawn means the drug was pulled from the market or from development.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, Structure with Ligand, High-Quality Ligand, Druggable Family
- Antibody
- GO CC high conf, UniProt loc med conf, UniProt SigP or TMHMM
- PROTAC
- Small Molecule Binder
- Other modalities
- Advanced Clinical
gnomAD loss of function constraint: o/e 0.7091599702835083 (upper bound 1.0636999607086182). 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 11 registry trials, updated in the last three years, of the most advanced drugs against GHSR (macimorelin, ibutamoren, anamorelin, relamorelin, ulimorelin, PF-5190457, capromorelin, TZP-102, tabimorelin), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Sriram YennuM.D. Anderson Cancer Center · 2 of 2 trials Phase 2+
- Jose M GarciaUniversity of Washington and Veterans Affairs Puget Sound Health Care System · 1 of 1 trials Phase 2+
- Keith StuartLahey Hospital & Medical Center · 1 of 1 trials Phase 2+
- Laura E. DichtelMassachusetts General Hospital · 1 of 1 trials Phase 2+
- Nicola K AmmerAEterna Zentaris · 1 of 1 trials Phase 2+
- Sandy EisenNorgine Ltd · 1 of 1 trials Phase 2+
- Bess Dawson-HughesTufts University · 0 of 1 trials Phase 2+
Registry trials of macimorelin, ibutamoren, anamorelin, relamorelin, ulimorelin, PF-5190457, capromorelin, TZP-102, tabimorelin 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; 6 of 10 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. 1 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
13 trials of drugs against GHSR were stopped early; 2 give no reason
Terminated, withdrawn and suspended interventional trials of macimorelin, ibutamoren, anamorelin, relamorelin, ulimorelin, PF-5190457, capromorelin, TZP-102, tabimorelin, 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 13 most recent of 13.
- terminated · 2025-11-17 · Lahey Clinic · Anamorelin Hydrochloride“The study closure was initiated by the financial sponsor and agreed upon by the site PI. The study is closing because of low enrollment and insufficient funds.”
- terminated · 2022-03-16 · National Institute on Alcohol Abuse and Alcoholism (NIAAA) · matched by the registry's drug synonyms“Early termination of the study due to the COVID-19 pandemic”
- terminated · 2021-12-22 · Allergan · Relamorelin“The Relamorelin program is being terminated solely based on a business decision.”
- terminated · 2021-12-15 · Allergan · Relamorelin 10 μg“The Relamorelin program is being terminated solely based on a business decision.”
- terminated · 2021-11-23 · Allergan · Relamorelin“The Relamorelin program is being terminated solely based on a business decision.”
- terminated · 2021-08-06 · Allergan · Relamorelin“The Relamorelin program is being terminated solely based on a business decision”
- terminated · 2021-07-29 · Allergan · Relamorelin“The Relamorelin program is being terminated solely based on a business decision.”
- terminated · 2018-07-17 · VA Office of Research and Development · Anamorelin HCl“poor recruitment”
- withdrawn · 2018-05-14 · University of Virginia · matched by the registry's drug synonyms“Could not obtain drug supply from manufacturer”
- terminated · 2016-09-23 · Motus Therapeutics, Inc. · matched by the registry's drug synonymsNo reason given
- terminated · 2016-01-20 · Merck Sharp & Dohme LLC · matched by the registry's drug synonymsNo reason given
- terminated · 2012-12-20 · Tranzyme, Inc. · 10 mg TZP-102“Insufficient efficacy in planned interim futility analysis”
- terminated · 2012-05-23 · Pfizer · matched by the registry's drug synonyms“B3301002 was discontinued on 18 April 2012 for strategic reasons.There were no safety concerns leading to discontinuation of this study.”
Paediatric incentives and obligations for GHSR's drugs
For macimorelin, ibutamoren, anamorelin, relamorelin, ulimorelin, PF-5190457, capromorelin, TZP-102, tabimorelin: 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 · GHSR 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-001988-PIP01-16-M02 (macimorelin, Diagnosis of growth hormone deficiency): PM: decision on the application for modification of an agreed PIP, 13 Sept 2024; EMEA-003032-PIP01-21-M01 (Ibutamoren mesilate, Treatment of growth hormone deficiency): PM: decision on the application for modification of an agreed PIP, 10 Mar 2023; EMEA-002323-PIP02-19 (relamorelin, Treatment of gastroparesis): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 9 Sept 2020; EMEA-001365-PIP01-12 (anamorelin hydrochloride, Treatment of cancer related cachexia): W: decision granting a waiver in all age groups for all conditions or indications, 10 Jun 2014; +1 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/3/17/1882 positive 20 Jun 2017: Treatment of growth hormone deficiencyRule: 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/3/17/1882, 2026-11-11
- Authorised in the EU: yes · Ghryvelin (previously Macimorelin Aeterna Zentaris): authorised since 11 Jan 2019; Adlumiz: refusedRule: 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 macimorelin, ibutamoren, anamorelin, relamorelin, ulimorelin, pf-5190457, capromorelin, tzp-102, tabimorelinRule: 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 · 3 interventional studies of macimorelin, ibutamoren, anamorelin, relamorelin, ulimorelin, pf-5190457, capromorelin, tzp-102, tabimorelin include patients under 18; 1 is 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 12 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.