Disease · MONDO_0007037

achondroplasia

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. achondroplasia. Diseases, record MONDO_0007037. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0007037

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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: achondroplasia; Open Targets
Open Targets ID: MONDO_0007037; Open Targets
Other names: ACH; Open Targets
Disease Ontology: DOID:4480; Disease Ontology
MeSH: D000130; MeSH
NCI Thesaurus: C34345; NCI Thesaurus
Orphanet: ORPHA:15; Orphanet
Highest stage of its drugs: Approval, for 1 of its 6 drugs, from EMA Human Drugs, FDA, PMDA and TTD; Open Targets

Drugs6

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
VOSORITIDE	CHEMBL3707276	Protein	Approval	EMA Human Drugs, FDA, PMDA and TTD	12	8	8	Drugs and targets
INFIGRATINIB	CHEMBL1852688	Small molecule	Phase 3	ClinicalTrials.gov	5	5	5	Drugs and targets
NAVEPEGRITIDE	CHEMBL4594507	Protein	Phase 2/3	ClinicalTrials.gov and TTD	8	7	7	Drugs and targets
RECIFERCEPT	CHEMBL4650420	Protein	Phase 2	ClinicalTrials.gov and TTD	4	2	2	Drugs and targets
LONAPEGSOMATROPIN	CHEMBL4298185	Protein	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
SOMATROPIN	CHEMBL1201621	Protein	Unknown	ClinicalTrials.gov	2	2	2	Drugs and targets

Targets6

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
GHR	Growth hormone receptor	2	Growth hormone receptor agonist	Phase 2	https://conductscience.org/library/genes/2690	Drugs and targets
FGFR1	Fibroblast growth factor receptor	1	Fibroblast growth factor receptor inhibitor	Phase 3	https://conductscience.org/library/genes/2260	Drugs and targets
FGFR2	Fibroblast growth factor receptor	1	Fibroblast growth factor receptor inhibitor	Phase 3	https://conductscience.org/library/genes/2263	Drugs and targets
FGFR3	Fibroblast growth factor receptor	1	Fibroblast growth factor receptor inhibitor	Phase 3	https://conductscience.org/library/genes/2261	Drugs and targets
FGFR4	Fibroblast growth factor receptor	1	Fibroblast growth factor receptor inhibitor	Phase 3	https://conductscience.org/library/genes/2264	Drugs and targets
NPR2	Atrial natriuretic peptide receptor 2	1	Atrial natriuretic peptide receptor B binding agent	Approval	https://conductscience.org/library/genes/4882	Drugs and targets

Trials24

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
NCT03197766	A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia	Phase 3	121	2022-03-02	Achondroplasia	VOSORITIDE	Its record names the disease	ClinicalTrials.gov
NCT03583697	A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia	Phase 2	75	2024-06-13	Achondroplasia	VOSORITIDE	Its record names the disease	ClinicalTrials.gov
NCT04638153	A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia	Phase 2	60	2024-02-15	Achondroplasia	RECIFERCEPT	Its record names the disease	ClinicalTrials.gov
NCT04085523	A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia	Phase 2	57	2024-03-07	Achondroplasia	NAVEPEGRITIDE	Its record names the disease	ClinicalTrials.gov
NCT02055157	A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia	Phase 2	35	2021-01-15	Achondroplasia	VOSORITIDE	Its record names the disease	ClinicalTrials.gov
NCT05116046	Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia	Phase 2	35	2024-02-08	Achondroplasia	RECIFERCEPT	Its record names the disease	ClinicalTrials.gov
NCT01516229	Special Survey for Long Term Application		395		Genetic Disorder, Achondroplasia	SOMATROPIN	Its record names the disease	ClinicalTrials.gov
NCT05145010	Extension Study of Infigratinib in Children With Achondroplasia (ACH)	Phase 2	325		Achondroplasia	INFIGRATINIB	Its record names the disease	ClinicalTrials.gov
NCT07441876	Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia	Phase 2/3	160		Achondroplasia	VOSORITIDE	Its record names the disease	ClinicalTrials.gov
NCT05929807	A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia	Phase 2/3	140		Achondroplasia	NAVEPEGRITIDE	Its record names the disease	ClinicalTrials.gov

1 to 10 of 24

See all 24 trials

Comparisons 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
BMN 111 - 15 μg/kg vs Placebo: deaths from any cause	0 of 60	0 of 61	121	NCT03197766	Trial comparisons
BMN 111 - 15 μg/kg vs Placebo: serious adverse events	3 of 60	4 of 61	121	NCT03197766	Trial comparisons
Vosoritide vs Placebo: deaths from any cause	1 of 43	0 of 32	75	NCT03583697	Trial comparisons
Vosoritide vs Placebo: serious adverse events	3 of 43	6 of 32	75	NCT03583697	Trial comparisons

Papers from its trials16

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
37823031	RESULT	1	PubMed
42370681	DERIVED	1	PubMed
42319555	DERIVED	1	PubMed
42144862	DERIVED	2	PubMed
41783511	DERIVED	1	PubMed
41247754	DERIVED	1	PubMed
39740666	DERIVED	2	PubMed
39555818	DERIVED	1	PubMed
39305160	DERIVED	2	PubMed
38685585	DERIVED	1	PubMed

1 to 10 of 16

See all 16 papers from its trials

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/MONDO_0007037

GET https://conductscience.org/api/diseases/v1/MONDO_0007037/trials?page=2

API reference For AI assistants