Last reviewed · How we verify
AEGR-733
AEGR-733 is a MTP inhibitor Small molecule drug developed by Aegerion Pharmaceuticals, Inc.. It is currently in Phase 3 development for Familial chylomicronemia syndrome (Type I hyperlipoproteinemia), Severe hypertriglyceridemia. Also known as: lomitapide, BMS-201038.
AEGR-733 is a microsomal triglyceride transfer protein (MTP) inhibitor that reduces the production and secretion of apolipoprotein B-containing lipoproteins.
AEGR-733 is a microsomal triglyceride transfer protein (MTP) inhibitor that reduces the production and secretion of apolipoprotein B-containing lipoproteins. Used for Familial chylomicronemia syndrome (Type I hyperlipoproteinemia), Severe hypertriglyceridemia.
-
Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Cardiovascular Phase 3 risk
-2.0pp
Modern cardiovascular outcome trials are large + long; many fail to beat aggressive standard-of-care.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | AEGR-733 |
|---|---|
| Also known as | lomitapide, BMS-201038 |
| Sponsor | Aegerion Pharmaceuticals, Inc. |
| Drug class | MTP inhibitor |
| Target | Microsomal triglyceride transfer protein (MTP) |
| Modality | Small molecule |
| Therapeutic area | Cardiovascular / Lipidology |
| Phase | Phase 3 |
Mechanism of action
By inhibiting MTP, AEGR-733 decreases the assembly and secretion of VLDL and chylomicrons from the liver and intestine, leading to reduced plasma levels of triglycerides and LDL cholesterol. This mechanism is particularly relevant for severe hypertriglyceridemia and familial chylomicronemia syndrome, where elevated triglycerides pose a risk of acute pancreatitis.
Approved indications
- Familial chylomicronemia syndrome (Type I hyperlipoproteinemia)
- Severe hypertriglyceridemia
Common side effects
- Gastrointestinal effects (diarrhea, steatorrhea, abdominal discomfort)
- Elevated liver transaminases
- Fat-soluble vitamin deficiency
Key clinical trials
- Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia
- Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH) (PHASE3)
- LOWER: Lomitapide Observational Worldwide Evaluation Registry
- Global Lomitapide Pregnancy Exposure Registry
- Study to Determine the Intra-subject Variability of Pharmacokinetics of Lomitapide in Healthy Subjects (PHASE1)
- Evaluate the Effect of Atorvastatin on the Pharmacokinetics of Lomitapide in Healthy Subjects. (PHASE1)
- Evaluate the Effect of Ethinyl Estradiol/Norgestimate on the Pharmacokinetics of Lomitapide in Healthy Female Subjects (PHASE1)
- Phase I Study of the Safety, Tolerability, PK & PD of Lomitapide in Japanese and Caucasian Subjects With Elevated LDL-C (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- AEGR-733 CI brief — competitive landscape report
- AEGR-733 updates RSS · CI watch RSS
- Aegerion Pharmaceuticals, Inc. portfolio CI
Frequently asked questions about AEGR-733
What is AEGR-733?
How does AEGR-733 work?
What is AEGR-733 used for?
Who makes AEGR-733?
Is AEGR-733 also known as anything else?
What drug class is AEGR-733 in?
What development phase is AEGR-733 in?
What are the side effects of AEGR-733?
What does AEGR-733 target?
Related
- Drug class: All MTP inhibitor drugs
- Target: All drugs targeting Microsomal triglyceride transfer protein (MTP)
- Manufacturer: Aegerion Pharmaceuticals, Inc. — full pipeline
- Therapeutic area: All drugs in Cardiovascular / Lipidology
- Indication: Drugs for Familial chylomicronemia syndrome (Type I hyperlipoproteinemia)
- Indication: Drugs for Severe hypertriglyceridemia
- Also known as: lomitapide, BMS-201038
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing