Disease · MONDO_0018637

familial chylomicronemia syndrome

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. familial chylomicronemia syndrome. Diseases, record MONDO_0018637. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0018637

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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: familial chylomicronemia syndrome; Open Targets
Open Targets ID: MONDO_0018637; Open Targets
Disease Ontology: DOID:0111417; Disease Ontology
Orphanet: ORPHA:444490; Orphanet
Highest stage of its drugs: Approval, for 1 of its 5 drugs, from ClinicalTrials.gov; Open Targets

Drugs5

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
OLEZARSEN	CHEMBL4650438	Oligonucleotide	Approval	ClinicalTrials.gov	5	4	4	Drugs and targets
PLOZASIRAN	CHEMBL6068438	Oligonucleotide	Phase 3	ClinicalTrials.gov and TTD	4	3	2	Drugs and targets
PRADIGASTAT	CHEMBL2364624	Small molecule	Phase 3	ClinicalTrials.gov and TTD	4	3	3	Drugs and targets
VOLANESORSEN	CHEMBL3544994	Oligonucleotide	Phase 3	ClinicalTrials.gov	4	3	3	Drugs and targets
VUPANORSEN	CHEMBL4298197	Oligonucleotide	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets4

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
APOC3	Apolipoprotein C-III mRNA	2	Apolipoprotein C-III mRNA 3'UTR antisense inhibitor; Apolipoprotein C-III mRNA antisense inhibitor	Approval	https://conductscience.org/library/genes/345	Drugs and targets
ANGPTL3	Angiopoietin like 3 (ANGPTL3) mRNA	1	Angiopoietin like 3 (ANGPTL3) mRNA antisense inhibitor	Phase 2	https://conductscience.org/library/genes/27329	Drugs and targets
ASGR1	Asialoglycoprotein receptor 1	1	Asialoglycoprotein receptor 1 binding agent	Phase 2	https://conductscience.org/library/genes/432	Drugs and targets
DGAT1	Diacylglycerol O-acyltransferase 1	1	Diacylglycerol O-acyltransferase 1 inhibitor	Phase 3	https://conductscience.org/library/genes/8694	Drugs and targets

Trials13

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
NCT05089084	Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)	Phase 3	75	2026-02-04	Familial Chylomicronemia	PLOZASIRAN	Its record names the disease	ClinicalTrials.gov
NCT02658175	The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome	Phase 3	68	2021-08-26	Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency, Hyperlipoproteinemia Type 1	VOLANESORSEN	Its record names the disease	ClinicalTrials.gov
NCT02211209	The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome	Phase 3	67	2022-04-13	Familial Chylomicronemia Syndrome	VOLANESORSEN	Its record names the disease	ClinicalTrials.gov
NCT04568434	A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)	Phase 3	66	2025-03-06	Familial Chylomicronemia Syndrome	OLEZARSEN	Its record names the disease	ClinicalTrials.gov
NCT01514461	A Randomized, Double-blind, Placebo Controlled Study to Assess Efficacy, Safety and Tolerability of LCQ908 in Subjects With Familial Chylomicronemia Syndrome	Phase 3	45	2015-06-03	Familial Chylomicronemia Syndrome (FCS)	PRADIGASTAT	Its record names the disease	ClinicalTrials.gov
NCT01589237	Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome.	Phase 3	38	2016-11-15	Familial Chylomicronemia Syndrome (FCS) (HLP Type I)	PRADIGASTAT	Its record names the disease	ClinicalTrials.gov
NCT03360747	Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)	Phase 2	3	2021-01-07	Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency, Hyperlipoproteinemia Type 1	VUPANORSEN	Its record names the disease	ClinicalTrials.gov
NCT05130450	A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)	Phase 3	60		Familial Chylomicronemia Syndrome	OLEZARSEN	Its record names the disease	ClinicalTrials.gov
NCT05185843	A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen	Phase 3	24		Familial Chylomicronemia Syndrome	OLEZARSEN	Its record names the disease	ClinicalTrials.gov
NCT01146522	Safety, Tolerability,Pharmacokinetics(PK)and Pharmacodynamics(PD)Assessment of LCQ908 in Patients With Severe Hypertriglyceridemia	Phase 1/2	8		Hyperlipoproteinemia	PRADIGASTAT	Its record names the disease	ClinicalTrials.gov

1 to 10 of 13

See all 13 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
Volanesorsen vs Placebo: Treatment Response Rate Defined as Participants With Fasting TG ≥ 40% Reduction From Baseline at Month 3	29 of 33	3 of 33	66	NCT02211209	Trial comparisons
Volanesorsen vs Placebo: deaths from any cause	0 of 33	0 of 33	66	NCT02211209	Trial comparisons
Volanesorsen vs Placebo: serious adverse events	7 of 33	5 of 33	66	NCT02211209	Trial comparisons
Volanesorsen vs Placebo: Treatment Response Rate Defined as Participants With Fasting Plasma TG < 750 mg/dL at Month 3	23 of 30	3 of 31	61	NCT02211209	Trial comparisons

Papers from its trials9

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
38587247	RESULT	1	PubMed
31390500	RESULT	1	PubMed
41923352	DERIVED	1	PubMed
41545246	DERIVED	1	PubMed
39225259	DERIVED	1	PubMed
37164837	DERIVED	1	PubMed
29748148	DERIVED	1	PubMed
27271183	DERIVED	1	PubMed
25889044	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/MONDO_0018637

GET https://conductscience.org/api/diseases/v1/MONDO_0018637/drugs

API reference For AI assistants