Product Description
Mipomersen is a novel antisense oligonucleotide inhibitor of apolipoprotein B. It is approved by the FDA only as an orphan drug for use in HoFH, a relatively rare genetic condition. (Sourced from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3956393/)
Mechanisms of Action: APOB Inhibitor
Novel Mechanism: No
Modality: Nucleic Acid
Route of Administration: Subcutaneous
FDA Designation: None *
Approval Status: Approved
Approved Countries: Dominican Republic | European Medicines Agency | Peru
Approved Indications: Hypercholesterolemia
Known Adverse Events: Headache
Company: Kastle
Company Location:
Company CEO:
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count:
Highest Development Phases
Phase 3: Hyperlipoproteinemia Type II|Lipid Metabolism Disorders|Lipid Metabolism, Inborn Errors|Hypercholesterolemia|Hyperlipoproteinemias|Dyslipidemia|Hyperlipidemia|Coronary Artery Disease|Myocardial Ischemia|Atherosclerosis
Phase 2: Dyslipidemia|Hypercholesterolemia|Hyperlipoproteinemias|Hyperlipoproteinemia Type II|Hyperlipidemia|Lipid Metabolism Disorders|Hypolipoproteinemias|Lipid Metabolism, Inborn Errors|Hypobetalipoproteinemias|Atherosclerosis
Phase 1: Healthy Volunteers|Hypercholesterolemia|Coronary Disease
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| FOCUS FH | P3 |
Completed |
Hypercholesterolemia |
2016-03-15 |
|
| FOCUS FH | P3 |
Completed |
Hypercholesterolemia|Hyperlipoproteinemia Type II |
2015-12-29 |
|
| MICA | P2 |
Terminated |
Hypercholesterolemia|Atherosclerosis |
2015-05-15 |
|
| MICA | P3 |
Completed |
Atherosclerosis|Hypercholesterolemia |
2015-05-01 |