FGFR1 fibroblast growth factor receptor 1
ENSG00000077782 · protein coding · Open Targets profile
Tyrosine-protein kinase that acts as a cell-surface receptor for fibroblast growth factors and plays an essential role in the regulation of embryonic development, cell proliferation, differentiation and migration. Required for normal mesoderm patterning and correct axial organization during embryonic development, normal skeletogenesis and normal development of the gonadotropin-releasing hormone (GnRH) neuronal system. Phosphorylates PLCG1, FRS2, GAB1 and SHB. Ligand binding leads to the activation of several signaling cascades. Activation of PLCG1 leads to the production of the cellular signaling molecules diacylglycerol and inositol 1,4,5-trisphosphate. Phosphorylation of FRS2 triggers recruitment of GRB2, GAB1, PIK3R1 and SOS1, and mediates activation of RAS, MAPK1/ERK2, MAPK3/ERK1 and the MAP kinase signaling pathway, as well as of the AKT1 signaling pathway. Promotes phosphorylation of SHC1, STAT1 and PTPN11/SHP2. In the nucleus, enhances RPS6KA1 and CREB1 activity and contributes to the regulation of transcription. FGFR1 signaling is down-regulated by IL17RD/SEF, and by FGFR1 ubiquitination, internalization and degradation. {ECO:0000250|UniProtKB:P16092, ECO:0000269|PubMed:10830168, ECO:0000269|PubMed:11353842, ECO:0000269|PubMed:12181353, ECO:0000269|PubMed:1379697, ECO:0000269|PubMed:1379698, ECO:0000269|PubMed:15117958, ECO:0000269|PubMed:16597617, ECO:0000269|PubMed:17311277, ECO:0000269|PubMed:17623664, ECO:0000269|PubMed:18480409, ECO:0000269|PubMed:19224897, ECO:0000269|PubMed:19261810, ECO:0000269|PubMed:19665973, ECO:0000269|PubMed:20133753, ECO:0000269|PubMed:20139426, ECO:0000269|PubMed:21765395, ECO:0000269|PubMed:8622701, 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 Withdrawn means the drug was pulled from the market or from development.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
- Approved Drug, Structure with Ligand, High-Quality Ligand, High-Quality Pocket, Druggable Family
- Antibody
- GO CC high conf, UniProt loc med conf, UniProt SigP or TMHMM
- PROTAC
- Literature, UniProt Ubiquitination, Database Ubiquitination, Half-life Data, Small Molecule Binder
- Other modalities
- Advanced Clinical
gnomAD loss of function constraint: o/e 0.19205999374389648 (upper bound 0.2847999930381775). 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 400 registry trials, updated in the last three years, of the most advanced drugs against FGFR1 (E-7090, nintedanib esylate, infigratinib, surufatinib, pemigatinib, futibatinib, pazopanib, erdafitinib, nintedanib, regorafenib, brivanib, derazantinib), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Peter LangmuirIncyte Corporation · 4 of 4 trials Phase 2+
- William SchelmanHutchmed · 3 of 4 trials Phase 2+
- Jihui HaoTianjin Medical University Cancer Institute and Hospital · 3 of 3 trials Phase 2+Ph2NCT05908747 · Efficacy and Safety of Surufatinib Combined With Gemcitabine and Albumin-bound Paclitaxel in the Peri-operative Treatment of Pancreatic Cancerbrief
- Jean-Yves BLAYCentre Leon Berard · 2 of 3 trials Phase 2+Ph2NCT02029001 · Adapting Treatment to the Tumor Molecular Alterations for Patients With Advanced Solid Tumors: MyOwnSpecificTreatmentbrief
- Marwan G FakihCity of Hope Medical Center · 2 of 3 trials Phase 2+Ph1/Ph2NCT05672316 · Botensilimab, Balstilimab and Regorafenib for the Treatment of Patients With Microsatellite Stable Metastatic Colorectal Cancer Who Have Progressed on Prior Chemotherapybrief
- Alain C MitaECOG-ACRIN Cancer Research Group · 2 of 2 trials Phase 2+
- Hui LiuSun yat-sen universtiy cancer center · 2 of 2 trials Phase 2+
- Julien EdelineCentre Eugène Marquis · 2 of 2 trials Phase 2+
- Kanwal RaghavM.D. Anderson Cancer Center · 2 of 2 trials Phase 2+
- Lin ShenPeking University Cancer Hospital & Institute · 2 of 2 trials Phase 2+
- Nicolas PENELCentre Oscar Lambret · 2 of 2 trials Phase 2+
- Song GaoTianjin Medical University Cancer Institute and Hospital · 2 of 2 trials Phase 2+
- Wenjing JiangAnhui Provincial Cancer Hospital · 2 of 2 trials Phase 2+
- Ying YuanSecond Affiliated Hospital, Zhejiang University, School of Medicine · 2 of 2 trials Phase 2+
- Florence DUFFAUDLa Timone University Hospital · 1 of 2 trials Phase 2+
Registry trials of E-7090, nintedanib esylate, infigratinib, surufatinib, pemigatinib, futibatinib, pazopanib, erdafitinib, nintedanib, regorafenib, brivanib, derazantinib 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; 281 of 370 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. 30 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
186 trials of drugs against FGFR1 were stopped early; 27 give no reason
Terminated, withdrawn and suspended interventional trials of E-7090, nintedanib esylate, infigratinib, surufatinib, pemigatinib, futibatinib, pazopanib, erdafitinib, nintedanib, regorafenib, brivanib, derazantinib, 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 186.
- terminated · 2026-11-10 · National Institute of Dental and Craniofacial Research (NIDCR) · matched by the registry's drug synonyms“Study was stopped early due to a greater than expected incidence of ocular AEs and analysis of the data from the first 4 subjects indicating that the likelihood of permanent remission with this therapy was low.”
- terminated · 2026-11-03 · Peking University Cancer Hospital & Institute · regorafenib“The study was stopped early due to lack of efficacy.”
- suspended · 2026-10-20 · City of Hope Medical Center · Regorafenib“resources”
- terminated · 2026-10-05 · Children's Oncology Group · Regorafenib“Drug supply issues”
- terminated · 2026-08-26 · Bristol-Myers Squibb · RegorafenibNo reason given
- terminated · 2026-08-19 · Boundless Bio, Inc. · Futibatinib“Decision was made to halt the study based on overall clinical experience and market considerations. This decision was not driven by any safety signal.”
- terminated · 2026-08-11 · Incyte Corporation · Pemigatinib“The study was terminated due to lack of enrollment resulting from a change in the standard of care for the first-line treatment of patients with cholangiocarcinoma. There were no safety concerns that contributed to this decision.”
- terminated · 2026-07-06 · Syndax Pharmaceuticals · matched by the registry's drug synonyms“Study was terminated by sponsor after Phase 1b as pre-defined signals of efficacy were not met in the Phase 1b dose expansion cohort.”
- withdrawn · 2026-06-15 · University of Miami · Regorafenib“Investigator decision”
- terminated · 2026-06-11 · Changhai Hospital · Surufatinib“This investigator-initiated study closed to recruitment early due to slower-than-anticipated patient accrual.”
- terminated · 2026-04-14 · Institut Claudius Regaud · Pazopanib“Slow recruitment rate”
- terminated · 2026-03-27 · M.D. Anderson Cancer Center · Futibatinib“PI request”
- withdrawn · 2026-03-11 · M.D. Anderson Cancer Center · Regorafenib“0 participants enrolled”
- terminated · 2026-03-09 · Translational Research in Oncology · Regorafenib in combination with pembrolizumab“The trial ended earlier than planned due to slow enrolment, which has made it unfeasible to achieve the target sample size within a reasonable timeframe. Consequently, a business decision has been made to discontinue funding for the trial.”
- terminated · 2026-03-05 · Wake Forest University Health Sciences · Regorafenib“Slow accruals”
- withdrawn · 2026-02-25 · M.D. Anderson Cancer Center · Regorafenib“0 participants enrolled”
- terminated · 2026-02-12 · Jennifer Lee Caswell-Jin · Infigratinib“Drug manufacturer decision to terminate development.”
- terminated · 2026-02-06 · M.D. Anderson Cancer Center · Erdafitinib“<75% participation”
- terminated · 2026-01-20 · Anwaar Saeed · Regorafenib“Terminated per directive from Bayer due to funding.”
- terminated · 2026-01-13 · Novartis Pharmaceuticals · matched by the registry's drug synonyms“Sponsor Decision”
- terminated · 2025-12-31 · Taiho Oncology, Inc. · Futibatinib“Sponsor decided to discontinue the study due to strategic considerations and not due to any safety-related concerns.”
- terminated · 2025-12-30 · Incyte Corporation · Pemigatinib“Recruitment ceased after a pre-planned futility interim analysis indicated a low probability to confer a clinically meaningful improvement in objective response when compared to currently available therapies. There were no safety related concerns.”
- terminated · 2025-11-13 · Taiho Oncology, Inc. · Futibatinib, Futibatinib plus Fulvestrant“The Sponsor decided to discontinue the study due to strategic considerations and not due to any safety-related concerns.”
- terminated · 2025-11-04 · Incyte Corporation · Pemigatinib“A business decision was made to to discontinue further enrollment. There were no safety concerns that contributed to this decision.”
- terminated · 2025-11-04 · Incyte Corporation · Pemigatinib“The reason this study was terminated was due to a business decision. There were no safety concerns that contributed to this decision.”
Paediatric incentives and obligations for FGFR1's drugs
For E-7090, nintedanib esylate, infigratinib, surufatinib, pemigatinib, futibatinib, pazopanib, erdafitinib, nintedanib, regorafenib, brivanib, derazantinib: 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 · FGFR1 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 · EMA/PE/0000223498 (Infigratinib, Treatment of achondroplasia): PM: decision on the application for modification of an agreed PIP, 3 Jan 2025; EMA/PE/0000182170 (regorafenib, Treatment of all conditions contained in the category of malignant neoplasms (except haematopoietic and lymphoid tissue)): PM: decision on the application for modification of an agreed PIP, 5 Dec 2024; EMEA-002042-PIP03-23 (Erdafitinib, Treatment of urothelial carcinoma): W: decision granting a waiver in all age groups for all conditions or indications, 6 May 2024; EMEA-002370-PIP03-22 (pemigatinib, Treatment of myeloid/lymphoid neoplasms with eosinophilia and gene rearrangement): W: decision granting a waiver in all age groups for all conditions or indications, 1 Dec 2022; +11 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/3/21/2475 positive 19 Jul 2021: Treatment of achondroplasia; EU/3/19/2146 withdrawn 1 Apr 2019: Treatment of biliary tract cancer; EU/3/25/3157 positive 21 Nov 2025: Treatment of hypochondroplasia; EU/3/19/2177 withdrawn 28 Jun 2019: Treatment of glioma; +7 moreRule: 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 · Pemazyre: authorised since 26 Mar 2021, orphan medicine, conditional; Ofev: authorised since 14 Jan 2015; Nintedanib Accord: authorised since 19 Apr 2024; Balversa: authorised since 22 Aug 2024; +7 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 e-7090, nintedanib esylate, infigratinib, surufatinib, pemigatinib, futibatinib, pazopanib, erdafitinib, nintedanib, regorafenib, brivanib, derazantinibRule: 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 · 57 interventional studies of e-7090, nintedanib esylate, infigratinib, surufatinib, pemigatinib, futibatinib, pazopanib, erdafitinib, nintedanib, regorafenib, brivanib, derazantinib include patients under 18; 23 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, 2026-11-11
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 26 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.