FKBP1A FKBP prolyl isomerase 1A
ENSG00000088832 · protein coding · Open Targets profile
Keeps in an inactive conformation TGFBR1, the TGF-beta type I serine/threonine kinase receptor, preventing TGF-beta receptor activation in absence of ligand. Recruits SMAD7 to ACVR1B which prevents the association of SMAD2 and SMAD3 with the activin receptor complex, thereby blocking the activin signal. May modulate the RYR1 calcium channel activity. PPIases accelerate the folding of proteins. It catalyzes the cis-trans isomerization of proline imidic peptide bonds in oligopeptides. {ECO:0000269|PubMed:16720724, ECO:0000269|PubMed:1696686, ECO:0000269|PubMed:1701173, ECO:0000269|PubMed:9233797}.
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 Withdrawn means the drug was pulled from the market or from development.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
- UniProt loc med conf
- PROTAC
- Literature, Database Ubiquitination, Half-life Data, Small Molecule Binder
gnomAD loss of function constraint: o/e 0.3533099889755249 (upper bound 0.7428799867630005). 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 FKBP1A (zotarolimus, temsirolimus, tacrolimus anhydrous, pimecrolimus, everolimus, sirolimus), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Christopher G KanakryNational Cancer Institute (NCI) · 3 of 4 trials Phase 2+Ph1/Ph2NCT05436418 · The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantationbrief
- Elizabeth M KangNational Institute of Allergy and Infectious Diseases (NIAID) · 3 of 4 trials Phase 2+Early Ph1NCT03910452 · Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamidebrief
- Monzr M Al MalkiCity of Hope Medical Center · 3 of 4 trials Phase 2+Ph2NCT04262843 · Total Marrow and Lymphoid Irradiation as Conditioning Regimen Before Hematopoietic Cell Transplantation in Patients With Myelodysplastic Syndrome or Acute LeukemiabriefPh2NCT05364762 · Adding Itacitinib to Cyclophosphamide and Tacrolimus for the Prevention of Graft Versus Host Disease in Patients Undergoing Hematopoietic Stem Cell TransplantsbriefPh2NCT07582172 · Total Marrow and Lymphoid Irradiation in Combination With Fludarabine and Melphalan as Conditioning for Allogeneic Peripheral Blood Stem Cell Hematopoietic Cell Transplant in Older Patients With Refractory and Relapsed Acute Myeloid Leukemia and High-risk Myelodysplastic Syndromebrief
- Damiano RondelliUniversity of Illinois Chicago · 3 of 3 trials Phase 2+Ph2NCT03121014 · Study of Intensity Modulated Total Marrow Irradiation (IM-TMI) in Addition to Fludarabine/Busulfan Conditioning for Allogeneic Transplantation in High Risk AML and Myelodysplastic Syndromesbrief
- Kenneth NewellEmory University · 3 of 3 trials Phase 2+
- Marie BleakleyFred Hutch/University of Washington Cancer Consortium · 3 of 3 trials Phase 2+Ph2NCT03970096 · Graft Versus Host Disease-Reduction Strategies for Donor Blood Stem Cell Transplant Patients With Acute Leukemia or Myelodysplastic Syndrome (MDS)brief
- Uday R PopatM.D. Anderson Cancer Center · 3 of 3 trials Phase 2+
- Akshay SharmaSt. Jude Children's Research Hospital · 2 of 2 trials Phase 2+
- Bhavisha A PatelNational Cancer Institute (NCI) · 2 of 2 trials Phase 2+
- Christian P. LarsenEmory University · 2 of 2 trials Phase 2+
- Danielle E Pregent-ArnoldNational Cancer Institute (NCI) · 2 of 2 trials Phase 2+
- Dimana DimitrovaNational Cancer Institute (NCI) · 2 of 2 trials Phase 2+
- Flavio VincentiUniversity of California at San Francisco · 2 of 2 trials Phase 2+
- Gary ShangoldSponsor CMO · 2 of 2 trials Phase 2+
- Haris AliCity of Hope Medical Center · 2 of 2 trials Phase 2+
Registry trials of zotarolimus, temsirolimus, tacrolimus anhydrous, pimecrolimus, everolimus, sirolimus 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; 312 of 384 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. 16 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
578 trials of drugs against FKBP1A were stopped early; 8 of the 200 most recent give no reason
Terminated, withdrawn and suspended interventional trials of zotarolimus, temsirolimus, tacrolimus anhydrous, pimecrolimus, everolimus, sirolimus, 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 578.
- suspended · 2026-11-10 · National Cancer Institute (NCI) · Sirolimus“Administrative hold pending non-safety related changes to the study design.”
- terminated · 2026-11-05 · M.D. Anderson Cancer Center · Temsirolimus“< 75 % participation”
- terminated · 2026-10-22 · Odense University Hospital · matched by the registry's drug synonyms“Insufficient drug supply (prasugrel)”
- suspended · 2026-10-15 · City of Hope Medical Center · matched by the registry's drug synonyms“Timeline Adjustment”
- withdrawn · 2026-10-15 · Brigham and Women's Hospital · matched by the registry's drug synonyms“Attempts to renew our protocol have not been accepted. Also, composition of the team and institutional goals have changed.”
- terminated · 2026-09-24 · National Institute of Allergy and Infectious Diseases (NIAID) · matched by the registry's drug synonyms“Slow accrual within enrollment time period: projected accrual goal not achieved.”
- terminated · 2026-09-24 · National Institute of Allergy and Infectious Diseases (NIAID) · matched by the registry's drug synonyms“Absence of equipoise on the basis of predetermined stopping rules.”
- terminated · 2026-09-24 · National Institute of Allergy and Infectious Diseases (NIAID) · matched by the registry's drug synonyms“Secondary to safety concerns plus change in Campath® (alemtuzumab) availability.”
- terminated · 2026-09-24 · National Institute of Allergy and Infectious Diseases (NIAID) · matched by the registry's drug synonyms“Safety: Stopping rule not met.”
- terminated · 2026-09-24 · National Institute of Allergy and Infectious Diseases (NIAID) · matched by the registry's drug synonyms“Due to inability to meet accrual goals within the funding period”
- suspended · 2026-09-10 · Leland Metheny · matched by the registry's drug synonyms“insufficient funding”
- withdrawn · 2026-09-03 · Mayo Clinic · matched by the registry's drug synonyms“Sponsor declined to fund study due to inability to agree on IP.”
- terminated · 2026-07-29 · Vanderbilt University Medical Center · matched by the registry's drug synonyms“PI departure from institution”
- terminated · 2026-07-27 · M.D. Anderson Cancer Center · Temsirolimus“<75% participation”
- terminated · 2026-07-22 · M.D. Anderson Cancer Center · Everolimus“<75% participation”
- withdrawn · 2026-07-07 · Peking Union Medical College Hospital · sirolimus and ATRA, sirolimus“The study has not actually been conducted.”
- terminated · 2026-07-07 · M.D. Anderson Cancer Center · matched by the registry's drug synonyms“Significant Adverse Effects - Futility”
- terminated · 2026-06-18 · M.D. Anderson Cancer Center · matched by the registry's drug synonyms“Toxicity and Lack of Efficacy”
- terminated · 2026-06-10 · Columbia University · Sirolimus Oral Tablet“Closed by the industry collaborator”
- terminated · 2026-06-04 · Institut National de la Santé Et de la Recherche Médicale, France · everolimus“results from interim analysis conducted to study interruption”
- terminated · 2026-05-27 · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · matched by the registry's drug synonyms“The stopping rule was met and hence the study was closed”
- terminated · 2026-05-14 · Nader Sanai · Temsirolimus“Study terminated due to unavailability of the investigational product required to continue the study.”
- terminated · 2026-05-14 · Fred Hutchinson Cancer Center · Sirolimus“Study was terminated prior to enrolling protocol target accrual goal due to declining accrual and Data Safety Monitoring Board recommendations.”
- terminated · 2026-05-06 · Mats Remberger · matched by the registry's drug synonyms“Too slow recruitment”
- terminated · 2026-05-06 · Nicholas Butowski · matched by the registry's drug synonyms“Drug no longer provided by sponsor”
Paediatric incentives and obligations for FKBP1A's drugs
For zotarolimus, temsirolimus, tacrolimus anhydrous, pimecrolimus, everolimus, sirolimus: 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 · FKBP1A 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-003168-PIP01-21 (sirolimus, Treatment of tuberous sclerosis complex): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 2 Dec 2022; EMEA-003168-PIP01-21 (sirolimus, Treatment of tuberous sclerosis complex): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 2 Dec 2022; EMEA-002982-PIP01-21 (sirolimus, Treatment of ornithine transcarbamylase deficiency): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 10 Jun 2022; EMEA-001416-PIP01-12-M03 (sirolimus, Treatment of chronic non-infectious uveitis): PM: decision on the application for modification of an agreed PIP, 11 Aug 2021; +8 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/3/25/3189 positive 9 Jan 2026: Treatment of osteosarcoma; EU/3/25/3051 positive 16 Apr 2025: Treatment of familial adenomatous polyposis (FAP); EU/3/10/764 expired 4 Aug 2010: Treatment of tuberous sclerosis; EU/3/13/1204 positive 13 Nov 2013: Prevention of arteriovenous access dysfunction in haemodialysis patients; +17 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 · Afinitor: authorised since 2 Aug 2009; Hyftor: authorised since 15 May 2023, orphan medicine; Rapamune: authorised since 13 Mar 2001; Votubia: authorised since 2 Sept 2011; +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: yes · Everolimus [Afinitor] &, 29 Oct 2010: Adult and pediatric patients aged 1 year and older with tuberous sclerosis complex (TSC) for the treatment of subependymal giant cell astrocytoma (SEGA) that requires therapeutic intervention but cannot be curatively resectedRule: 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 · 768 interventional studies of zotarolimus, temsirolimus, tacrolimus anhydrous, pimecrolimus, everolimus, sirolimus include patients under 18; 164 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 7 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.