Hypercholesterolemia
Recent clinical, regulatory, research and industry developments relating to this disease.
Small Interfering RNA to Reduce Lipoprotein(a) in Cardiovascular Disease.
Two Phase 3 Trials of Inclisiran in Patients with Elevated LDL Cholesterol.
Childhood adiposity, adult adiposity, and cardiovascular risk factors.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 6 clinical trials expected to report results, the earliest in Q4 2027.
- Q4 2027A Phase Ⅲ Randomized, Parallel-group, Placebo-controlled Trial to Assess the Efficacy, Safety of the SYH2053 Subcutaneous Injection as Monotherapy in Participants With Primary Hypercholesterolemia (Non-familial) or Mixed Dyslipidemia
- Q1 2028Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children (6 to Less Than 12 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL- Cholesterol
- Q1 2028Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Children (2 to Less Than 12 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol
- Q4 2028A Phase 3, Open-label Extension Study to Evaluate the Safety and Efficacy of MK-0616 in Adults With Hypercholesterolemia
- Q1 2032An Open-label, Single Arm, Multicenter Extension Study to Evaluate Long-term Safety and Tolerability of Inclisiran in Participants With Heterozygous or Homozygous Familial Hypercholesterolemia Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20 or ORION-19 Studies (VICTORION-PEDS-OLE)
Clinical MilestonesView all 11Hide
- 2026-07-10An Operationally Seamless Phase 2/3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Enlicitide Decanoate in Pediatric Participants With Heterozygous Familial HypercholesterolemiaResults expected Q4 2033
- 2026-05-15An Open-label, Single Arm, Multicenter Extension Study to Evaluate Long-term Safety and Tolerability of Inclisiran in Participants With Heterozygous or Homozygous Familial Hypercholesterolemia Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20 or ORION-19 Studies (VICTORION-PEDS-OLE)Results expected Q1 2032
- 2026-05-15Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children (6 to Less Than 12 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL- CholesterolResults expected Q1 2028
- 2026-04-07A Phase Ⅲ Randomized, Parallel-group, Placebo-controlled Trial to Assess the Efficacy, Safety of the SYH2053 Subcutaneous Injection as Monotherapy in Participants With Primary Hypercholesterolemia (Non-familial) or Mixed DyslipidemiaResults expected Q4 2027
- 2026-02-20Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Children (2 to Less Than 12 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterolResults expected Q1 2028
- 2026-05-12A Multicenter, Randomized, Double-blinded, Active-controlled, Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of AG2304T and AG2304R in Patients With Primary HypercholesterolemiaCompleted
- 2026-01-13A Real-world, Prospective, Multi-center, Open-label, Phase 4 Clinical Study to Evaluate the Safety and Effectiveness of Subcutaneous Injection of Inclisiran Sodium in Indian Patients With Primary Hypercholesterolemia or Mixed DyslipidemiaCompleted
- 2026-01-02A Randomized, Multicenter, Open-label Trial Comparing the Effectiveness of Inclisiran to Bempedoic Acid on LDL Cholesterol (LDL-C) Lowering in Participants With Atherosclerotic Cardiovascular Disease (VICTORION-CHALLENGE)Completed
- 2025-11-18A Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Clinical Study Evaluating the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial HypercholesterolemiaCompleted
- 2025-11-11A Multicenter, Randomized, Double-blind, Active-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Co-administrated BR1018B and BR1018C in Patients With Essential Hypertension and Primary HypercholesterolemiaCompleted
- 2026-07-10ClinicalAn Operationally Seamless Phase 2/3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Enlicitide Decanoate in Pediatric Participants With Heterozygous Familial HypercholesterolemiaResults expected Q4 2033
- 2026-05-15ClinicalAn Open-label, Single Arm, Multicenter Extension Study to Evaluate Long-term Safety and Tolerability of Inclisiran in Participants With Heterozygous or Homozygous Familial Hypercholesterolemia Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20 or ORION-19 Studies (VICTORION-PEDS-OLE)Results expected Q1 2032
- 2026-05-15ClinicalTwo Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children (6 to Less Than 12 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL- CholesterolResults expected Q1 2028
- 2026-05-12ClinicalA Multicenter, Randomized, Double-blinded, Active-controlled, Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of AG2304T and AG2304R in Patients With Primary HypercholesterolemiaCompleted
- 2026-04-07ClinicalA Phase Ⅲ Randomized, Parallel-group, Placebo-controlled Trial to Assess the Efficacy, Safety of the SYH2053 Subcutaneous Injection as Monotherapy in Participants With Primary Hypercholesterolemia (Non-familial) or Mixed DyslipidemiaResults expected Q4 2027
- 2026-02-20ClinicalTwo Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Children (2 to Less Than 12 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterolResults expected Q1 2028
- 2026-01-13ClinicalA Real-world, Prospective, Multi-center, Open-label, Phase 4 Clinical Study to Evaluate the Safety and Effectiveness of Subcutaneous Injection of Inclisiran Sodium in Indian Patients With Primary Hypercholesterolemia or Mixed DyslipidemiaCompleted
- 2026-01-02ClinicalA Randomized, Multicenter, Open-label Trial Comparing the Effectiveness of Inclisiran to Bempedoic Acid on LDL Cholesterol (LDL-C) Lowering in Participants With Atherosclerotic Cardiovascular Disease (VICTORION-CHALLENGE)Completed
- 2025-11-18ClinicalA Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Clinical Study Evaluating the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial HypercholesterolemiaCompleted
- 2025-11-11ClinicalA Multicenter, Randomized, Double-blind, Active-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Co-administrated BR1018B and BR1018C in Patients With Essential Hypertension and Primary HypercholesterolemiaCompleted
Clinical trials
The current development programme across all trial phases.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes8
- Anticholesteremic Agents1
- Atherosclerosis1
- Fermented Foods1
- Hypercholesterolemia1
- Lipoprotein(a)1
- PCSK9 Inhibitors1
- RNA, Small Interfering1
- Tea1
Leading journals4
- The New England journal of medicine3
- Circulation2
- European heart journal1
- Nature communications1
Leading researchers8
- Albus C1
- Baum SJ1
- Benlian P1
- Berenson GS1
- Bian Z1
- Bisch JA1
- Boysen G1
- Brewer HB Jr1
Affiliations (unnormalised)6
- Brigham and Women's Hospital and Harvard Medical School1
- Chinese Medicine Clinical Study Center1
- From the Imperial Centre for Cardiovascular Disease Prevention1
- Functional Metabolomic and Gut Microbiome Laboratory1
- Lung and Blood Institute's Framingham Heart Study1
- Research Center of Applied and Preventive Cardiovascular Medicine Department of Medicine1
Disease biology
Key proteins & gene products studied in this disease. Number shows shared papers.
Related conditions
Diseases frequently studied alongside this one. Number shows shared papers.
Disease profile
A grounded synthesis of the condition — overview, causes, mechanism, risk factors and current standard of care.
Hypercholesterolemia is a condition in which cholesterol levels in the blood are abnormally high. In the supplied definition, it is identified by a cholesterol value above the 95th percentile for the population.
The condition is characterized by elevated circulating cholesterol, including low-density lipoprotein cholesterol (LDL-C) as a treatment target in the supplied literature. The grounding also links the topic to cholesterol, LDL, and small interfering RNA, but does not provide a specific disease mechanism beyond abnormal blood cholesterol elevation.
Higher body mass index is associated with cardiovascular risk factors in the supplied literature, and consistently high adiposity from childhood into adulthood is linked to increased risk of adverse cardiovascular risk profiles. The grounding does not provide hypercholesterolemia-specific risk factors beyond this association.
Management includes therapeutic lifestyle changes and cholesterol-lowering drug therapy. The supplied review abstracts specifically support statin-based cholesterol-lowering therapy in high-risk patients and reference LDL-C as a treatment goal, with diabetes included among high-risk categories in the guideline context.
AI-generated summary grounded in MeSH and 2 peer-reviewed sources. Informational only — not medical advice. Generated 2026-07-07.
Reference
Authoritative identity, definition & identifiers.
A condition with abnormally high levels of CHOLESTEROL in the blood. It is defined as a cholesterol value exceeding the 95th percentile for the population.
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.