Disease · MONDO_0001676
erythropoietic protoporphyria
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. erythropoietic protoporphyria. Diseases, record MONDO_0001676. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0001676
On this page
Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Highest stage of its drugs
- Approval, for 1 of its 11 drugs, from DailyMed, EMA Human Drugs and FDA
- Open Targets
Drugs11
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| AFAMELANOTIDE | CHEMBL441738 | Protein | Approval | DailyMed, EMA Human Drugs and FDA | 9 | 6 | 6 | Drugs and targets |
| BITOPERTIN | CHEMBL1171829 | Small molecule | Phase 3 | ClinicalTrials.gov | 5 | 5 | 5 | Drugs and targets |
| DERSIMELAGON | CHEMBL4802160 | Small molecule | Phase 3 | ClinicalTrials.gov and TTD | 6 | 4 | 4 | Drugs and targets |
| COLESTIPOL HYDROCHLORIDE | CHEMBL1201474 | Phase 2/3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets | |
| CIMETIDINE | CHEMBL30 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| FERRIC CARBOXYMALTOSE | CHEMBL2108597 | Unknown | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets | |
| FERRIC OXYHYDROXIDE | CHEMBL4802224 | Small molecule | Unknown | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| FERROUS SULFATE | CHEMBL1200830 | Small molecule | Unknown | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| IRON ISOMALTOSIDE 1000 | CHEMBL4298046 | Unknown | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets | |
| ISONIAZID | CHEMBL64 | Small molecule | Unknown | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
1 to 10 of 11
See all 11 drugsTargets3
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| MC1R | Melanocyte-stimulating hormone receptor | 2 | Melanocortin receptor 1 agonist | Approval | https://conductscience.org/library/genes/4157 | Drugs and targets |
| HRH2 | Histamine H2 receptor | 1 | Histamine H2 receptor antagonist | Phase 2 | https://conductscience.org/library/genes/3274 | Drugs and targets |
| SLC6A9 | Sodium- and chloride-dependent glycine transporter 1 | 1 | Glycine transporter 1 inhibitor | Phase 3 | https://conductscience.org/library/genes/6536 | Drugs and targets |
Trials18
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 1 more is not shown, since the checks do not confirm it.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT04402489 | Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria | Phase 3 | 184 | 2025-03-21 | EPP, XLP | DERSIMELAGON | Its record names the disease | ClinicalTrials.gov |
| NCT03520036 | Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria | Phase 2 | 102 | 2023-01-20 | Erythropoietic Protoporphyria (EPP) | DERSIMELAGON | Its record names the disease | ClinicalTrials.gov |
| NCT04053270 | Multicentre Phase III Erythropoietic Protoporphyria Study | Phase 3 | 100 | 2019-10-10 | Erythropoietic Protoporphyria | AFAMELANOTIDE | Its record names the disease | ClinicalTrials.gov |
| NCT01605136 | Phase III Confirmatory Study in Erythropoietic Protoporphyria | Phase 3 | 93 | 2019-08-26 | Erythropoietic Protoporphyria | AFAMELANOTIDE | Its record names the disease | ClinicalTrials.gov |
| NCT01097044 | Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP) | Phase 2 | 77 | 2019-09-17 | Erythropoietic Protoporphyria | AFAMELANOTIDE | Its record names the disease | ClinicalTrials.gov |
| NCT05308472 | Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP | Phase 2 | 75 | 2026-01-08 | Erythropoietic Protoporphyria | BITOPERTIN | Its record names the disease | ClinicalTrials.gov |
| NCT00979745 | Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP) | Phase 3 | 74 | 2021-10-12 | Erythropoietic Protoporphyria | AFAMELANOTIDE | Its record names the disease | ClinicalTrials.gov |
| NCT04578496 | A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP) | Phase 3 | 16 | 2020-11-02 | Erythropoietic Protoporphyria | AFAMELANOTIDE | Its record names the disease | ClinicalTrials.gov |
| NCT01550705 | Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic Protoporphyria | Not applicable | 11 | 2017-01-16 | Erythropoietic Protoporphyria (EPP), X Linked Erythropoietic Protoporphyria | ISONIAZID | Its record names the disease | ClinicalTrials.gov |
| NCT01422915 | Sorbent Therapy of the Cutaneous Porphyrias | Phase 2/3 | 4 | 2017-04-25 | Erythropoietic Protoporphyria | COLESTIPOL HYDROCHLORIDE | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 18
See all 18 trialsComparisons in its trials4
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| Afamelanotide vs Placebo: serious adverse events | 3 of 100 | 3 of 100 | 200 | NCT04053270 | Trial comparisons |
| Afamelanotide vs Placebo: serious adverse events | 3 of 48 | 2 of 45 | 93 | NCT01605136 | Trial comparisons |
| Afamelanotide vs Placebo: serious adverse events | 2 of 39 | 2 of 38 | 77 | NCT01097044 | Trial comparisons |
| Experimental: Afamelanotide vs Placebo: serious adverse events | 0 of 38 | 0 of 36 | 74 | NCT00979745 | Trial comparisons |
Papers from its trials4
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0001676
GET https://conductscience.org/api/diseases/v1/MONDO_0001676/drugs