IDH1 isocitrate dehydrogenase (NADP(+)) 1
ENSG00000138413 · protein coding · Open Targets profile
Catalyzes the NADP(+)-dependent oxidative decarboxylation of isocitrate (D-threo-isocitrate) to 2-ketoglutarate (2-oxoglutarate), which is required by other enzymes such as the phytanoyl-CoA dioxygenase (PubMed:10521434, PubMed:19935646). Plays a critical role in the generation of NADPH, an important cofactor in many biosynthesis pathways (PubMed:10521434). May act as a corneal epithelial crystallin and may be involved in maintaining corneal epithelial transparency (By similarity). {ECO:0000250|UniProtKB:Q9XSG3, ECO:0000269|PubMed:10521434, ECO:0000269|PubMed:19935646, ECO:0000303|PubMed:10521434}.
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 Withdrawn means the drug was pulled from the market or from development.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
- Structure with Ligand, High-Quality Ligand, High-Quality Pocket, Approved Drug
- Antibody
- GO CC high conf
- PROTAC
- Database Ubiquitination, Half-life Data, Small Molecule Binder
gnomAD loss of function constraint: o/e 0.7160400152206421 (upper bound 0.9574400186538696). 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 100 registry trials, updated in the last three years, of the most advanced drugs against IDH1 (olutasidenib, ivosidenib, vorasidenib, safusidenib, DS-1001B, IDH305), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Courtney DiNardoM.D. Anderson Cancer Center · 5 of 6 trials Phase 2+Ph1/Ph2NCT06445959 · Phase 1b/2 Study of Decitabine and Venetoclax in Combination With the Targeted Mutant IDH1 Inhibitor Olutasidenibbrief
- Julien EdelineCentre Eugène Marquis · 2 of 2 trials Phase 2+
- Kelly ChienM.D. Anderson Cancer Center · 2 of 2 trials Phase 2+
- Amir T FathiMassachusetts General Hospital · 1 of 2 trials Phase 2+
- Alice MimsBeat AML · 1 of 1 trials Phase 2+
- Alice S MimsOhio State University Comprehensive Cancer Center · 1 of 1 trials Phase 2+
- Arnaud PIGNEUXUniversity Hospital, Bordeaux · 1 of 1 trials Phase 2+
- B.J. WoutersErasmus MC / HOVON · 1 of 1 trials Phase 2+Ph3NCT03839771 · A Study of Ivosidenib or Enasidenib in Combination With Induction Therapy and Consolidation Therapy, Followed by Maintenance Therapy in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myedysplastic Syndrome EB2, With an IDH1 or IDH2 Mutation, Respectively, Eligible for Intensive Chemotherapybrief
- Brian JonasUniversity of California, Davis · 1 of 1 trials Phase 2+
- Bristol-Myers SquibbBristol-Myers Squibb · 1 of 1 trials Phase 2+Ph1/Ph2NCT02677922 · A Study to Assess the Safety and Efficacy of Two Combinations of Isocitrate Dehydrogenase (IDH) Mutant Targeted Therapies Plus Azacitidine in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) Harboring IDH Mutations Who Are Not Candidates to Receive Intensive Induction Chemotherapybrief
- Ciara M KellyMemorial Sloan Kettering Cancer Center · 1 of 1 trials Phase 2+
- Donald W ParsonsChildren's Oncology Group · 1 of 1 trials Phase 2+
- Elizabeth D AlvaChildren's Oncology Group · 1 of 1 trials Phase 2+
- Emma BarrettForma Therapeutics · 1 of 1 trials Phase 2+
- Giovanni MartinelliUniversity of Bologna · 1 of 1 trials Phase 2+
Registry trials of olutasidenib, ivosidenib, vorasidenib, safusidenib, DS-1001B, IDH305 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; 60 of 94 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. 6 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
13 trials of drugs against IDH1 were stopped early; 1 give no reason
Terminated, withdrawn and suspended interventional trials of olutasidenib, ivosidenib, vorasidenib, safusidenib, DS-1001B, IDH305, BAY1436032, 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.
- withdrawn · 2026-10-29 · Virginia Commonwealth University · Olutasidenib Investigational Agent Administration“Budgetary constraints”
- withdrawn · 2026-10-05 · M.D. Anderson Cancer Center · Olutasidenib“PI request”
- terminated · 2026-06-01 · Northwestern University · Ivosidenib“Low accrual”
- terminated · 2026-04-20 · Servier Bio-Innovation LLC · Ivosidenib“The sponsor decided to terminate the study during the safety lead-in phase.”
- withdrawn · 2026-01-28 · Duke University · matched by the registry's drug synonyms“sponsor withdrew funding”
- withdrawn · 2024-07-31 · University of Chicago · Ivosidenib“unable to accrue”
- terminated · 2024-05-06 · Academic and Community Cancer Research United · Ivosidenib“Low accrual”
- withdrawn · 2022-06-09 · City of Hope Medical Center · Ivosidenib“Drug availability”
- withdrawn · 2022-01-20 · Yale University · matched by the registry's drug synonyms“No patients were able to be screened nor enrolled.”
- withdrawn · 2020-07-07 · Vanderbilt-Ingram Cancer Center · matched by the registry's drug synonyms“Loss of funding”
- withdrawn · 2019-04-10 · Novartis Pharmaceuticals · IDH305No reason given
- withdrawn · 2017-12-20 · NYU Langone Health · IDH305“Novartis paused all study start-up activities due to safety evaluation of IDH305 compound.”
- withdrawn · 2017-10-11 · University of Texas Southwestern Medical Center · IDH305“Sponsor did not want to move forward with protocol; study was never opened.”
Paediatric incentives and obligations for IDH1's drugs
For olutasidenib, ivosidenib, vorasidenib, safusidenib, DS-1001B, IDH305, BAY1436032: 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 · IDH1 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-003421-PIP01-23 (olutasidenib, Treatment of acute myeloid leukaemia): W: decision granting a waiver in all age groups for all conditions or indications, 29 Dec 2023; EMEA-002247-PIP03-17-M01 (Ivosidenib, Treatment of acute myeloid leukaemia): PM: decision on the application for modification of an agreed PIP, 3 Jan 2024; EMEA-002247-PIP02-17 (Ivosidenib, Treatment of all conditions included in the category of malignant neoplasms (except central nervous system tumours, haematopoietic and lymphoid tissue neoplasms);Treatment of malignant neoplasms of the central nervous system): RP: decision refers to a refusal on a proposed Paediatric Investigation Plan, 4 Dec 2019Rule: 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/3/25/3085 positive 18 Jul 2025: Treatment of myelodysplastic syndromes; EU/3/24/2978 positive 10 Nov 2024: Treatment of chondrosarcoma; EU/3/18/1994 positive 21 Mar 2018: Treatment of biliary tract cancer; EU/3/16/1802 positive 12 Dec 2016: Treatment of acute myeloid leukaemia; +2 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 · Voranigo: authorised since 17 Sept 2025, orphan medicine; Tibsovo: authorised since 4 May 2023, orphan medicine; Tidhesco: application withdrawn, orphan medicine; Tibsovo: application withdrawnRule: 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 · Vorasidenib [Voranigo], 6 Aug 2024: Treatment of adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 (IDH1) or isocitrate dehydrogenase-2 (IDH2) mutation following surgery including biopsy, sub-total resection, or gross total resectionRule: 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 · 12 interventional studies of olutasidenib, ivosidenib, vorasidenib, safusidenib, ds-1001b, idh305, bay1436032 include patients under 18; 12 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 8 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.