mipomersen (Discontinued)

Brand and Other Names:Kynamro
  • Print

Dosing & Uses


Dosage Forms & Strengths

subcutaneous injection

  • 200mg/mL

Homozygous Familial Hypercholesterolemia

Indicated as an adjunct to lipid-lowering medications and diet to reduce LDL-C, apoB, TC, and non-HDL-C in patients with homozygous familial hypercholesterolemia (HoFH)

200 mg SC once weekly

Dosage Modifications

Elevated transaminases

  • ≥3x and <5x ULN: Confirm by repeating measurement within 1 week; if confirmed withhold dosing
  • ≥5x ULN: Withhold dosing
  • Obtain additional liver-related tests if not already measured (eg, total bilirubin, alkaline phosphatase, INR) and investigate to identify the probable cause
  • Recommendations are based on ULN of ~ 30-40 IU/L
  • If transaminase elevations accompanied by clinical symptoms of liver injury (eg, nausea, vomiting, abdominal pain, fever, jaundice, lethargy, flu-like symptoms), increases in bilirubin ≥2x ULN, or active liver disease, discontinue treatment and investigate to identify the probable cause
  • If resuming mipomersen after transaminases resolve to <3x ULN consider monitoring liver-related tests more frequently

Dosing Considerations

Measure baseline transaminases (ALT, AST), alkaline phosphatase, and total bilirubin

Safety and effectiveness not been established in patients with hypercholesterolemia who do not have HoFH

Effect on cardiovascular morbidity and mortality undetermined

Safety and effectiveness of mipomersen as an adjunct to LDL apheresis have not been established; therefore, the use as an adjunct to LDL apheresis is not recommended

Monitor lipid levels at least q3months during the first year of treatment to determine if the LDL-C reduction achieved is sufficiently robust to warrant the potential risk of liver toxicity; maximal LDL-C reduction observed after ~6 months

Safety and efficacy not established



Interaction Checker

and mipomersen

No Results

     activity indicator 
    No Interactions Found
    Interactions Found


      Serious - Use Alternative

        Significant - Monitor Closely


            All Interactions Sort By:
             activity indicator 

            Adverse Effects


            Injection site reactions (84%)

            Increased hepatic fat >5% (62%)

            Fatigue (15%)

            Nausea (14%)

            Influenza-like illness (13%)

            Headache (12%)


            ALT increased (10%)

            Pyrexia (8%)

            Hypertension (7%)

            Hepatic steatosis (7%)

            Extremity pain (7%)

            AST increased (6%)

            Chills (6%)

            Abnormal LFTs (5%)

            Edema (5%)

            Musculoskeletal pain (4%)

            Vomiting (4%)

            Angina (4%)

            Hepatic enzyme increased (3%)

            Abdominal pain (3%)

            Palpitations (3%)

            Insomnia (3%)

            Postmarketing Reports

            Idiopathic thrombocytopenic purpura



            Black Box Warnings

            In the clinical trial, 4 of 34 (12%) patients treated with mipomersen had at least 1 elevation in ALT ≥3x ULN compared with 0% of the 17 patients treated with placebo

            Measure ALT, AST, alkaline phosphatase, and total bilirubin before initiating treatment and then ALT, AST regularly as recommended

            During treatment, withhold dosing if ALT or AST are ≥3 x ULN

            Discontinue clinically significant liver toxicity

            Increases hepatic steatosis with or without concomitant increases in transaminases

            Hepatic steatosis associated with therapy; may be a risk factor for progressive liver disease, including steatohepatitis and cirrhosis

            Because of risk of hepatotoxicity, therapy is available only through a restricted REMS program; should only be prescribed to patients with a clinical or laboratory diagnosis consistent with HoFH; safety and efficacy in patients with hypercholesterolemia who do not have HoFH not established



            Moderate-to-severe hepatic impairment (Child-Pugh B or C) or active liver disease, including unexplained persistent increased serum transaminases


            May cause elevations in transaminases and hepatic steatosis (see Black Box Warnings, Dosage Modifications); concern exists that these increases could induce steatohepatitis, which can progress to cirrhosis over several years

            Infection site reactions reported

            Women of reproductive potential should use effective contraception during mipomersen therapy

            Not recommended with severe renal impairment, clinically significant proteinuria, or on renal dialysis (lack of clinical data)

            Site reactions, including erythema, pain, tenderness, pruritus and local swelling reported

            Flu-like symptoms, occurring within 2 days after injection reported; symptoms may include influenza-like illness, pyrexia, chills, myalgia, arthralgia, malaise or fatigue

            Generalized hypersensitivity (eg, angioedema, urticaria, generalized rash) reported; if hypersensitivity occurs, instruct patient to promptly seek medical advice regarding discontinuation


            Pregnancy & Lactation

            Pregnancy Category: B

            Lactation: Unknown whether distributed in breast milk

            Pregnancy Categories

            A: Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.

            B: May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk.

            C: Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done.

            D: Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk.

            X: Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist.

            NA: Information not available.



            Mechanism of Action

            Antisense oligonucleotide inhibitor that targets messenger RNA for apolipoprotein B-100, the principal apolipoprotein of LDL and its metabolic precursor, VLDL


            Bioavailability: 54-78%

            Peak plasma time: 3-4 hr


            Protein bound: ≥90%

            Distribution half-life: 2-5 hr

            Steady-state typically reached within 6 months


            Metabolized in tissues by endonucleases to form shorter oligonucleotides that are then substrates for additional metabolism by exonucleases


            Half-life: 1-2 months

            Excretion: <4% urine over 24 hr



            SC Preparation

            Allowed drug to reach room temperature for at least 30 minutes before administering

            Do not use if solution is cloudy or contains visible particulate matter

            SC Administration

            For subcutaneous use only; do not administer IM or IV

            Administer on the same day each week; if a dose is missed, the injection should be given at least 3 days from the next weekly dose

            Inject SC into abdomen, thigh region, or outer area of the upper arm

            Do not inject in areas of active skin disease or injury (eg, sunburns, skin rashes, inflammation, skin infections, active areas of psoriasis)

            Avoid areas of tattooed skin and scarring


            Vials or syringes: Store refrigerated between 2-8°C (36-46°F)



            Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.