Rosuvastatin
| Evidence Level: L5 | Predicted Indications: 6 |
Table of Contents
Rosuvastatin: Cholesterol-ester transfer protein deficiency
One-Sentence Summary
Rosuvastatin is a statin (HMG-CoA reductase inhibitor) originally used for hypertriglyceridemia, hyperlipoproteinemia, atherosclerosis, stroke-related disorders and homozygous familial hypercholesterolemia. The top-ranked prediction is cholesterol-ester transfer protein deficiency, which has no registered clinical trials. The only genuinely novel prediction with a body of evidence is HIV infectious disease (a research question, not a recommendation).
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Not recorded in the local data |
| Predicted New Indication | Cholesterol-ester transfer protein deficiency |
| TxGNN Prediction Score | 0.9954 |
| Evidence Level | L5 for rank 1; L1 for Familial Hypercholesterolemia |
| India Market Status | Listed in a compiled Indian medicine price dataset (at least 20) – CDSCO approval status is not included in this data |
| Number of Listed Products | at least 20 |
| Recommended Decision | Hold for rank 1; Proceed with Guardrails for Familial Hypercholesterolemia |
Why is This Prediction Reasonable?
Detailed mechanism of action data is not available in the data compiled for this report. Based on known pharmacology, rosuvastatin is a statin that inhibits HMG-CoA reductase, reducing hepatic cholesterol synthesis and upregulating LDL receptors, which lowers LDL-C and triglycerides.
Rank 1 (cholesterol-ester transfer protein deficiency): This is a model prediction only, with no trials or publications. CETP deficiency raises HDL-C and alters lipoprotein metabolism, and no direct mechanistic or clinical link to rosuvastatin is documented. It should be treated as a hypothesis.
Familial hypercholesterolemia: The LDL clearance deficit in this condition is directly addressed by statin-induced LDL receptor upregulation. Homozygous familial hypercholesterolemia is already among the original indications, so this is mostly an established use. Guardrails: pediatric dosing, monitoring for myopathy and liver enzymes, and limited response in homozygous patients with null LDLR variants, who often need add-on therapy.
Other predictions in the list:
- Hyperlipidemia: an established use, with large Phase 3 programmes.
- HIV infectious disease (rank 5, L2): the rationale is lipid-lowering and anti-inflammatory effects reducing cardiovascular risk in people with HIV. The trials mostly address surrogate cardiovascular endpoints and drug interactions with antiretrovirals, not HIV itself, and results are mixed.
- Brain stem infarction, hypoalphalipoproteinemia, CYP7A1-deficiency hypercholesterolemia: only indirect or preclinical support.
- Hepatic lipase deficiency hyperlipidemia, ABri amyloidosis, a rare neurodevelopmental disorder: prediction only, no evidence.
Clinical Trial Evidence
Shown as a known use (up to 10 most relevant trials). The rank 1 prediction, CETP deficiency, has no registered clinical trials.
| Trial Number | Phase | Status | Enrollment | Key Findings |
|---|---|---|---|---|
| NCT00355615 | Phase 3 | Completed | 173 | Rosuvastatin vs placebo for 12 weeks, then 40 weeks open label, in children aged 10–17 with heterozygous FH; primary endpoint LDL-C reduction |
| NCT01078675 | Phase 3 | Completed | 315 | Open-label 2-year efficacy, safety and pharmacokinetic study in children and adolescents (6 to <18 years) with FH |
| NCT02226198 | Phase 3 | Completed | 20 | Double-blind, placebo-controlled crossover study in children and adolescents with homozygous FH |
| NCT02434497 | Phase 3 | Completed | 9 | Open-label extension evaluating long-term safety in paediatric homozygous FH |
| NCT00654602 | Phase 3 | Completed | 1500 | 48-week open-label study of efficacy and safety in Fredrickson type IIa/IIb dyslipidaemia, including heterozygous FH |
| NCT00654446 | Phase 3 | Completed | 442 | Renal effects of rosuvastatin vs simvastatin in type IIa/IIb dyslipidaemia, including heterozygous FH |
| NCT01709500 | Phase 3 | Completed | 249 | Alirocumab vs placebo in heterozygous FH on lipid-modifying therapy; rosuvastatin’s role (background therapy or test drug) is unclear |
| NCT03510884 | Phase 3 | Completed | 153 | Alirocumab in children with heterozygous FH on stable statin therapy; rosuvastatin’s role is unclear |
| NCT06910098 | N/A | Completed | 195 | Effect of different rosuvastatin doses on LDL, CPK and AST levels |
| NCT07474649 | Phase 3 | Not yet recruiting | 103 | Bempedoic acid/ezetimibe plus high-intensity atorvastatin or rosuvastatin on coronary plaque; no results yet |
Literature Evidence
Shown for Familial Hypercholesterolemia (up to 10 most relevant, RCTs first).
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 20223367 | 2010 | RCT | J Am Coll Cardiol | Efficacy and safety of rosuvastatin in children with familial hypercholesterolemia |
| 28838366 | 2017 | Cohort | J Am Coll Cardiol | Rosuvastatin efficacy in children with homozygous FH and its association with underlying genetic mutations |
| 26387811 | 2016 | Pharmacokinetic study | Eur J Clin Pharmacol | Population pharmacokinetics of rosuvastatin in paediatric heterozygous FH |
| 12269853 | 2002 | Review | Drugs | Rosuvastatin was superior to atorvastatin, simvastatin and pravastatin in improving lipid profile in hypercholesterolaemia across 6–52 week trials; low propensity for drug interactions |
| 15531000 | 2004 | Review | Clin Ther | Rosuvastatin in the management of hyperlipidemia, including homozygous FH |
| 28437620 | 2017 | Guideline | Endocr Pract | AACE/ACE guidelines for dyslipidemia management and cardiovascular prevention |
| 36928267 | 2023 | Cohort | J Atheroscler Thromb | Real-world LDL-C goal achievement in high-risk patients in Japan |
| 40254247 | 2025 | Mechanistic study | Toxicology | Statin myotoxicity in iPSC-derived muscle cells from FH patients, comparing lipophilic and hydrophilic statins |
| 20531065 | 2010 | Case report | South Med J | Rare severe thrombocytopenia during rosuvastatin treatment |
| 33976097 | 2021 | Review | Med Lett Drugs Ther | Evinacumab for homozygous FH |
India Market Information
| Authorization Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| 191412 | Rosutime 10mg Tablet (TTK Healthcare Ltd) | Strip of 10 tablets | Not specified |
| 193515 | Rosmi 5mg Tablet (Cadila Pharmaceuticals Ltd) | Strip of 10 tablets | Not specified |
| 36307 | Crevast 20 Tablet (Eris Lifesciences Ltd) | Strip of 15 tablets | Not specified |
| 184168 | Rosuvas 20 Tablet (Sun Pharmaceutical Industries Ltd) | Strip of 10 tablets | Not specified |
| 185654 | Rosave CV 20mg/75mg Capsule (Alembic Pharmaceuticals Ltd) | Strip of 10 capsules | Not specified |
All 20 registrations are valid. Several products carry rosuvastatin in combination with other agents (for example, the “CV” capsules), although the data classify all 20 as single-ingredient licences.
Safety Considerations
- Contraindications suggested by the knowledge graph (not from label text): liver disease, gallbladder disease, hypothyroidism, myxedema, congenital hypothyroidism, myopathy, muscular disease, skeletal muscle disease, familial periodic paralysis, substance abuse/dependence, rhabdomyolysis, nodular fasciitis, injury, proteinuria.
- Drug Interactions: 524 interactions are recorded. Major-level examples include fenofibrate and atazanavir. Moderate-level examples include atorvastatin, ezetimibe, bempedoic acid, amiodarone, paclitaxel, zidovudine, adalimumab, trastuzumab emtansine, alpelisib, apalutamide, asparaginase, auranofin, ethanol and sodium citrate. Minor-level examples include levonorgestrel, norethisterone and desogestrel.
- Literature signals: rare thrombocytopenia (case report), statin-associated incident diabetes, worsened insulin resistance in HIV-infected adults on rosuvastatin, and statin-associated muscle symptoms.
The package-insert warnings and contraindications for the Indian products have not been obtained. Please refer to the package insert for authoritative safety information.
Conclusion and Next Steps
Decision: Hold (CETP deficiency, the top-ranked prediction, and the other low-evidence predictions)
Rationale:
- The top-scored prediction, cholesterol-ester transfer protein deficiency (0.9954), has no trials or literature and rests on the model score alone.
- HIV infectious disease (L2) is worth investigating as a research question, but the evidence is mostly surrogate and mixed.
To proceed, the following is needed:
- Package-insert warnings and contraindications from the Indian regulatory source (a blocking gap for safety screening)
- Mechanism of action data from DrugBank
- Approved indication text for the Indian licences
- Paediatric dosing guidance, and a myopathy and liver-enzyme monitoring plan
- A review of the major drug interactions (fenofibrate, atazanavir) and dose adjustment in renal impairment and in Asian patients
- Any further evaluation of the HIV indication should focus on clinical outcomes rather than lipid or inflammatory surrogates
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.