FGF1 fibroblast growth factor 1
ENSG00000113578 · protein coding · Open Targets profile
Plays an important role in the regulation of cell survival, cell division, angiogenesis, cell differentiation and cell migration. Functions as a potent mitogen in vitro. Acts as a ligand for FGFR1 and integrins. Binds to FGFR1 in the presence of heparin leading to FGFR1 dimerization and activation via sequential autophosphorylation on tyrosine residues which act as docking sites for interacting proteins, leading to the activation of several signaling cascades. Binds to integrin ITGAV:ITGB3. Its binding to integrin, subsequent ternary complex formation with integrin and FGFR1, and the recruitment of PTPN11 to the complex are essential for FGF1 signaling. Induces the phosphorylation and activation of FGFR1, FRS2, MAPK3/ERK1, MAPK1/ERK2 and AKT1 (PubMed:18441324, PubMed:20422052). Can induce angiogenesis (PubMed:23469107). {ECO:0000269|PubMed:16597617, ECO:0000269|PubMed:18441324, ECO:0000269|PubMed:20145243, ECO:0000269|PubMed:20422052, ECO:0000269|PubMed:23469107, ECO:0000269|PubMed:8663044}.
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 Withdrawn means the drug was pulled from the market or from development.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
- Advanced Clinical, Structure with Ligand, High-Quality Ligand, Med-Quality Pocket, Druggable Family
- Antibody
- Advanced Clinical, GO CC high conf, UniProt loc med conf
- PROTAC
- Small Molecule Binder
- Other modalities
- Advanced Clinical
gnomAD loss of function constraint: o/e 0.6695700287818909 (upper bound 1.1684000492095947). 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 7 registry trials, updated in the last three years, of the most advanced drugs against FGF1 (suramin hexasodium, muparfostat, aldafermin, pegbelfermin, suramin, fazpilodemab), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Aram KimThompson Autism and Neurodevelopmental Center, Children's Hospital of Orange County · 1 of 1 trials Phase 2+
- jennifer L bonfriscoPaxmedica · 1 of 1 trials Phase 2+
- Stephen J RossiNGM Biopharmaceuticals, Inc · 1 of 1 trials Phase 2+
Registry trials of suramin hexasodium, muparfostat, aldafermin, pegbelfermin, suramin, fazpilodemab 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; 2 of 6 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.
5 trials of drugs against FGF1 were stopped early; 1 give no reason
Terminated, withdrawn and suspended interventional trials of suramin hexasodium, muparfostat, aldafermin, pegbelfermin, suramin, fazpilodemab, 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 5 most recent of 5.
- withdrawn · 2025-05-07 · NGM Biopharmaceuticals, Inc · aldafermin“Study was terminated by the Sponsor because the FDA advised a trial design change which made it infeasible to continue the study.”
- terminated · 2022-06-23 · Cellxpert Biotechnology Corp. · matched by the registry's drug synonyms“Due to Interim Analysis and business concerns”
- withdrawn · 2022-04-28 · Bristol-Myers Squibb · matched by the registry's drug synonyms“Business objectives have changed”
- terminated · 2015-05-21 · Optimum Therapeutics, LLC · Suramin Drug:Docetaxel Drug: CarboplatinNo reason given
- terminated · 2012-10-05 · SWOG Cancer Research Network · suramin“Permanently Closed Due to Lack of Accrual”
Paediatric incentives and obligations for FGF1's drugs
For suramin hexasodium, muparfostat, aldafermin, pegbelfermin, suramin, fazpilodemab: 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 · FGF1 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-003005-PIP01-21 (aldafermin, Treatment of non-alcoholic steatohepatitis (NASH)): W: decision granting a waiver in all age groups for all conditions or indications, 11 Aug 2021Rule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA EMEA-003005-PIP01-21, 2026-11-11
- EU orphan designation: no · No EU orphan designation names suramin hexasodium, muparfostat, aldafermin, pegbelfermin, suramin, fazpilodemabRule: 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: no · EMA lists no centrally authorised medicine of suramin hexasodium, muparfostat, aldafermin, pegbelfermin, suramin, fazpilodemabRule: 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 suramin hexasodium, muparfostat, aldafermin, pegbelfermin, suramin, fazpilodemabRule: 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 · 7 interventional studies of suramin hexasodium, muparfostat, aldafermin, pegbelfermin, suramin, fazpilodemab 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 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.