Disease · EFO_1002027

osteomalacia

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. osteomalacia. Diseases, record EFO_1002027. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/EFO_1002027

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.

Name: osteomalacia; Open Targets
Open Targets ID: EFO_1002027; Open Targets
Other names: adult osteomalacia, adult rickets, late rickets, rachitis tarda; Open Targets
MeSH: D010018; MeSH
Highest stage of its drugs: Approval, for 1 of its 2 drugs, from DailyMed, EMA Human Drugs and PMDA; Open Targets

Drugs2

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
BUROSUMAB	CHEMBL3707326	Antibody	Approval	DailyMed, EMA Human Drugs and PMDA	8	5	5	Drugs and targets
INFIGRATINIB	CHEMBL1852688	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets5

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
FGF23	Fibroblast growth factor 23	1	Fibroblast growth factor 23 inhibitor	Approval	https://conductscience.org/library/genes/8074	Drugs and targets
FGFR1	Fibroblast growth factor receptor	1	Fibroblast growth factor receptor inhibitor	Phase 2	https://conductscience.org/library/genes/2260	Drugs and targets
FGFR2	Fibroblast growth factor receptor	1	Fibroblast growth factor receptor inhibitor	Phase 2	https://conductscience.org/library/genes/2263	Drugs and targets
FGFR3	Fibroblast growth factor receptor	1	Fibroblast growth factor receptor inhibitor	Phase 2	https://conductscience.org/library/genes/2261	Drugs and targets
FGFR4	Fibroblast growth factor receptor	1	Fibroblast growth factor receptor inhibitor	Phase 2	https://conductscience.org/library/genes/2264	Drugs and targets

Trials6

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.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT02304367	Study of Burosumab (KRN23) in Adults With Tumor-Induced Osteomalacia (TIO) or Epidermal Nevus Syndrome (ENS)	Phase 2	17	2020-07-30	Tumor Induced Osteomalacia (TIO), Epidermal Nevus Syndrome (ENS)	BUROSUMAB	Its record names the disease	ClinicalTrials.gov
NCT02537431	Open Label Study of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)	Phase 3	14	2018-09-25	X-linked Hypophosphatemia	BUROSUMAB	Its record names the disease	ClinicalTrials.gov
NCT03510455	BGJ398 for the Treatment of Tumor-Induced Osteomalacia	Phase 2	4	2021-04-08	Tumor-Induced Osteomalacia, Oncogenic Osteomalacia	INFIGRATINIB	Open Targets' reading of its record	ClinicalTrials.gov
NCT06202027	Post Marketing Surveillance Study to Observe Safety and Effectiveness of CRYSVITA® in S. Korean Patients		100		FGF23-related Hypophosphataemic Rickets and Osteomalacia	BUROSUMAB	Its record names the disease	ClinicalTrials.gov
NCT04308096	A Study of KRN23 in Adult and Pediatric Patients With X-linked Hypophosphatemic Rickets/Osteomalacia	Phase 3	27		XLH	BUROSUMAB	Its record names the disease	ClinicalTrials.gov
NCT03233126	A Study of KRN23 in Pediatric Patients With X-linked Hypophosphatemic Rickets/Osteomalacia	Phase 3	16		X-linked Hypophosphatemic Rickets/Osteomalacia	BUROSUMAB	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials2

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
KRN23 TIO vs KRN23 XLH and ENS: deaths from any cause	2 of 14	0 of 3	17	NCT02304367	Trial comparisons
KRN23 TIO vs KRN23 XLH and ENS: serious adverse events	8 of 14	0 of 3	17	NCT02304367	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.

PMID	Listed as	Trials listing it	Source
39151033	DERIVED	1	PubMed
36719566	DERIVED	2	PubMed
35775373	DERIVED	1	PubMed
34660853	DERIVED	1	PubMed

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

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/EFO_1002027

GET https://conductscience.org/api/diseases/v1/EFO_1002027/drugs

API reference For AI assistants