Mipomersen

Orphan Drug Cold Chain RequiredFDA Approved

Description

Mipomersen is an antisense oligonucleotide inhibitor of apolipoprotein B-100 synthesis. It is indicated as an adjunct to lipid-lowering medications and diet to reduce LDL-C, apo B, total cholesterol, and non-HDL-C in patients with homozygous familial hypercholesterolemia (HoFH).

Indications & Therapeutic Use

Homozygous familial hypercholesterolemia

Global Availability (1 countries)

CountryAccess RouteRegulatory PathwayLead TimeStatus

Available Through

This drug is available for procurement through the following fulfilment partner.

Fulfilment Partner
Mipomersen
Generic NameMipomersen
Brands1 brand available
Active IngredientMipomersen sodium
Drug ClassHomozygous familial hypercholesterolemia
ManufacturerIonis Pharmaceuticals
Dosage FormsSubcutaneous injection, 200 mg/mL
Medical CodeC10AX13
Orphan StatusYes — Orphan Drug
Cold ChainRequired
Reg. StatusFDA Approved
Clinical TrialNCT00470875
Countries1 countries
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations2 Validated Nodes