DHODH dihydroorotate dehydrogenase (quinone)
ENSG00000102967 · protein coding · Open Targets profile
Catalyzes the conversion of dihydroorotate to orotate with quinone as electron acceptor. Required for UMP biosynthesis via de novo pathway. .
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 Withdrawn means the drug was pulled from the market or from development.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 SigP or TMHMM
- PROTAC
- Literature, Database Ubiquitination, Half-life Data, Small Molecule Binder
gnomAD loss of function constraint: o/e 0.7553600072860718 (upper bound 1.0245000123977661). 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 38 registry trials, updated in the last three years, of the most advanced drugs against DHODH (teriflunomide, vidofludimus, emvododstat, ASLAN-003, brequinar), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Xiao-Hui ZhangPeking University Institute of Hematology, Peking University People's Hospital · 4 of 4 trials Phase 2+
- Robert J. FoxMellen Center for MS, Neurological Institute, Cleveland Clinic, Ohio · 3 of 3 trials Phase 2+
- Andreas MuehlerImmunic AG · 2 of 2 trials Phase 2+
- Daniel OntanedaThe Cleveland Clinic · 1 of 1 trials Phase 2+
- Knut EA LundinOslo University Hospital · 1 of 1 trials Phase 2+
- Nathalie CoolsUniversiteit Antwerpen · 1 of 1 trials Phase 2+
- Nicolas COLLONGUESUniversity Hospital, Strasbourg, France · 1 of 1 trials Phase 2+
- Nikos EvangelouUniversity of Nottingham · 1 of 1 trials Phase 2+
- Pål-Dag LineOslo University Hoapital · 1 of 1 trials Phase 2+
- Roman IvanovJSC BIOCAD · 1 of 1 trials Phase 2+
- Steven JacobsonNational Institute of Neurological Disorders and Stroke (NINDS) · 1 of 1 trials Phase 2+
- Tatiana ScherzActelion · 1 of 1 trials Phase 2+
- Zwi BernemanUniversity Hospital, Antwerp · 1 of 1 trials Phase 2+
- Aurélie RUETUniversity Hospital, Bordeaux · 0 of 1 trials Phase 2+
- Ellen M. MowryJohns Hopkins University · 0 of 1 trials Phase 2+
Registry trials of teriflunomide, vidofludimus, emvododstat, ASLAN-003, brequinar 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; 18 of 38 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. Source: ClinicalTrials.gov, fetched live.
14 trials of drugs against DHODH were stopped early; 2 give no reason
Terminated, withdrawn and suspended interventional trials of teriflunomide, vidofludimus, emvododstat, ASLAN-003, brequinar, 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 14 most recent of 14.
- withdrawn · 2025-08-17 · University Hospital, Antwerp · matched by the registry's drug synonyms“Feasibility issues, insufficient funding, delays due transition to CTIS.”
- withdrawn · 2025-08-11 · Peking University People's Hospital · Teriflunomide“Trial withdrawn due to substantial protocol amendments invalidating the original design. With no participants enrolled, this aligns with WHO-ICTRP guidelines. Recruitment has ceased, and the revised protocol has been re-registered as NCT07065968.”
- terminated · 2025-06-12 · Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany · Teriflunomide“Primary analysis of the RMS phase 3 study (MS200527_0080) resulted in the early termination of the study.”
- terminated · 2025-03-21 · Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany · Teriflunomide“Primary analysis of the RMS phase 3 study (MS200527_0082) resulted in the early termination of the study.”
- terminated · 2024-03-05 · Immunic AG · matched by the registry's drug synonyms“Considering the efficacy results of the induction phase (allowed use of corticosteroids as concomitant medication) in the study population and since IMU-838 did not show superiority over placebo, the OLE was prematurely closed by the sponsor.”
- terminated · 2023-11-07 · Clear Creek Bio, Inc. · Brequinar Sodium“Low prevalence of COVID-19 in India led to lack of eligible subjects.”
- terminated · 2023-06-26 · PTC Therapeutics · matched by the registry's drug synonyms“Sponsor decision”
- terminated · 2022-08-08 · Clear Creek Bio, Inc. · Brequinar/Brequinar + Ribavirin“No efficacy observed, COVID-19 caused sites to shut down”
- terminated · 2022-02-02 · PTC Therapeutics · matched by the registry's drug synonyms“Sponsor terminated the study.”
- terminated · 2020-04-03 · Derrick Scott Robertson · teriflunomide“PK time points were not being met as expected. Determination that risks outweighed benefit.”
- terminated · 2019-05-10 · PTC Therapeutics · matched by the registry's drug synonymsNo reason given
- terminated · 2019-04-12 · PTC Therapeutics · matched by the registry's drug synonymsNo reason given
- terminated · 2018-06-06 · AIDS Malignancy Consortium · matched by the registry's drug synonyms“Drug supply unavailable.”
- terminated · 2014-06-09 · Sanofi · Teriflunomide, Placebo (for teriflunomide)“Sponsor decision to prematurely stop the study, not linked to any safety concern.”
Paediatric incentives and obligations for DHODH's drugs
For teriflunomide, vidofludimus, emvododstat, ASLAN-003, brequinar: 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 · DHODH 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-001094-PIP01-10-M04 (teriflunomide, Treatment of multiple sclerosis): PM: decision on the application for modification of an agreed PIP, 3 Jul 2017; compliance positive 23 Jun 2023Rule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA EMEA-001094-PIP01-10-M04, 2026-11-11
- EU orphan designation: no · No EU orphan designation names teriflunomide, vidofludimus, emvododstat, aslan-003, brequinarRule: 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 · Teriflunomide Viatris (previously Teriflunomide Mylan): authorised since 9 Nov 2022; Teriflunomide Accord: authorised since 9 Nov 2022; Aubagio: authorised since 26 Aug 2013Rule: 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 teriflunomide, vidofludimus, emvododstat, aslan-003, brequinarRule: 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 · 2 interventional studies of teriflunomide, vidofludimus, emvododstat, aslan-003, brequinar include patients under 18; 0 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 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.