Many patients with HoFH experience suboptimal responses with traditional, lipid-lowering therapies

Homozygous familial hypercholesterolemia (HoFH) is a rare and life-threatening disease.1
It is an inherited disorder of lipoprotein metabolism characterized by1:

  • Marked elevation of low-density lipoprotein-cholesterol (LDL-C) levels
  • Xanthomata
  • Premature cardiovascular disease

For these reasons, many patients with HoFH have suboptimal responses with traditional, lipid-lowering therapies, making treatment complex.2
KYNAMRO® (mipomersen sodium) injection, approved in January 2013, is adjunctive treatment that's added to your patients’ current lipid-lowering treatment regimen to help bring their LDL-C levels closer to target goals.3

FH Foundation

Kastle Therapeutics has partnered with the FH Foundation
to help raise awareness and improve the care of people living
with HoFH. Learn more

About HoFH

HoFH may increase plasma cholesterol

Genetic mutations that may cause HoFH severely impair LDL particle clearance, increasing plasma cholesterol.1

HoFH is a form of familial hypercholesterolemia (FH) which is caused by mutations in1:

  • LDL-receptor gene (LDL-R; >95%)
  • Apolipoprotein B (APO B; 2%-5%)
  • Pro-protein convertase subtilisin/kexin 9 (PCSK9; <1%)
  • Low density lipoprotein receptor adaptor protein 1 (LDLRAP1; <1%)
  • As-yet-unidentified genes

Diagnostic considerations of HoFH

Consider the following when diagnosing HoFH in your patients2-6:

  • Suboptimal responses to standard lipid-lowering therapies
  • Presence of premature heart disease
  • Family history, if known, of premature coronary heart disease and hypercholesterolemia in first-degree relatives
  • Physical findings of cholesterol deposition (corneal arcus, extensor tendon xanthomas, and/or planar or tuberous xanthomas); not present in every patient
  • Genetic heterogeneity
Genetic heterogeneity in HoFH chart Genetic heterogeneity in HoFH chart


Antisense Technology:
A targeted therapy for HoFH

KYNAMRO is the only therapy that uses antisense technology to inhibit the synthesis of apo B-100 by targeting a specific 20-base sequence on apo B-100 mRNA. KYNAMRO3:

  • Binds with the mRNA sequence and prevents the translation and formation of the protein apo B-100 in the hepatocyte
  • Inhibits the synthesis of apo B-100. KYNAMRO is designed to reduce the formation of very low-density lipoprotein (VLDL) and downstream atherogenic particles—including LDL
KYNAMRO (mipomersen sodium) Injection 200 mg/mL has Shown no clinically relevant interactions with common concomitant drugs KYNAMRO (mipomersen sodium) Injection 200 mg/mL has Shown no clinically relevant interactions with common concomitant drugs

*KYNAMRO has not been studied concomitantly with other LDL-lowering agents that can also increase hepatic fat; therefore, the combined use of such agents is not recommended.

Efficacy & Safety

Significant efficacy in the largest HoFH trial to date2,3

KYRAMRO (mipomersen sodium) Injection 200 mg/mL Efficacy vs Placebo KYRAMRO (mipomersen sodium) Injection 200 mg/mL Efficacy vs Placebo

Maximal reduction in LDL-C with KYNAMRO may take up to 6 months.

In a randomized, double-blind, placebo-controlled, 26-week trial in 51 subjects with HoFH§ (34 KYNAMRO, 17 placebo), KYNAMRO 200 mg/mL significantly reduced LDL-C and improved all other measured atherogenic lipoproteins when used as an adjunct to lipid-lowering medications and diet.2,3

  • 98% of subjects were taking a statin; 88% were on maximum-dose statin therapy2
  • Primary efficacy endpoint: percentage change in LDL-C from baseline to week 28, last observation carried forward3

Results are an average from the clinical trial. Individual results will vary. Absolute reduction based on the average baseline LDL-C of the study participants.2,3

113 mg/dL represents the absolute mean reduction in LDL-C from a baseline of 439 mg/dL .2

§Defined by the presence of at least one of the following: (1) history of genetic testing confirming 2 mutated alleles at the LDLR gene locus or (2) documented history of untreated LDL-C >500 mg/dL and at least one of the following: (a) tendinous and/or cutaneous xanthoma prior to age 10 years or (b) documentation of elevated LDL-C >190 mg/dL prior to lipid-lowering therapy consistent with heterozygous familial hypercholesterolemia (HeFH) in both parents. If a parent was not available, a history of coronary artery disease in a first-degree male relative of the parent younger than 55 years or first-degree female relative of the parent younger than 60 years was acceptable.3

See the profiles of the wide range of patients with HoFH who participated in the largest phase 3, placebo-controlled, clinical trial

Individual profiles with a clinical or laboratory diagnosis consistent with HoFH

In clinical trials the most commonly-reported adverse reactions were injection site reactions (84%), flu-like symptoms (30%), nausea (14%), headache (12%) and elevations in serum transaminases, specifically alanine aminotransferase (ALT) (10%).3

Adverse events should be reported to the Food & Drug Administration (FDA).
Visit or call 1-800-FDA-1088.

report adverse side effects report adverse side effects

LFT=liver function test.

Safety data are based on pooled results from 4 phase 3, randomized, double-blind, placebo-controlled trials with a total of 390 patients, of which 261 patients received weekly subcutaneous injections of 200 mg of KYNAMRO and 129 patients received placebo, for a median treatment duration of 25 weeks.3

Kynamro Dosing

KYNAMRO is available in a prefilled syringe that patients
self-inject once weekly3

KYNAMRO (mipomersen sodium) Injection 200 mg/mL Dosing KYNAMRO (mipomersen sodium) Injection 200 mg/mL Dosing

ALT=alanine aminotransferase;

AST=aspartate aminotransferase.

Download the Step-by-Step Administration
brochure to help patients learn how to self-inject

Prescribing KYNAMRO

Available through the KYNAMRO Risk Evaluation and Mitigation
Strategy (REMS) program

KYNAMRO is available only through a designated network of specialty pharmacies.

Only HCPs trained and enrolled in the KYNAMRO REMS program may prescribe KYNAMRO

The purpose of the KYNAMRO REMS program is to:

  • Educate prescribers about:
    • the risk of hepatotoxicity associated with the use of KYNAMRO
    • the need to monitor patients during treatment with KYNAMRO as per product labeling
  • Restrict access to therapy with KYNAMRO to patients with a clinical or laboratory diagnosis consistent with HoFH

Learn more

Train, enroll, and start prescribing

The materials needed to train and enroll in the KYNAMRO REMS program are available

Once complete, you may begin prescribing KYNAMRO for your patients with HoFH.

Train Icon

Train: Review all educational materials

Click here
Enroll Icon

Enroll: Download and complete the Prescriber Enrollment Form

Click here
Prescribe Icon

Prescribe: Download
and use the Prescription Authorization Form

Click here

Complete all forms and fax to KYNAMRO REMS at 1-877-778-9008.

KYNAMRO Cornerstone®

KYNAMRO Cornerstone logo

Personalized support
for you and your patients

KYNAMRO Cornerstone is a comprehensive support program that helps you and your patients with HoFH who have been prescribed KYNAMRO.

KYNAMRO Cornerstone Case Manager Icon

KYNAMRO Cornerstone
Case Manager
A dedicated partner who extends your
efforts to educate patients and keep them
taking KYNAMRO as you prescribed

KYNAMRO Access & Coverage Icon Access & Coverage
  • Assists with benefits verification, prior authorization, and coverage appeals
  • Helps find co-pay
    and financial assistance
    for patients who qualifyII
KYNAMRO Specialty Pharmacy Coordination Icon Specialty Pharmacy Coordination
  • Identifies a specialty pharmacy that works with your patients’ insurance
  • Coordinates the setup and delivery of medication to patients' homes or your office
KYNAMRO Self-injection Training Icon Self-injection Training
  • Facilitates scheduling
    of in-person, self-injection training for patients
    with a licensed nurse
KYNAMRO Ongoing Support Icon Ongoing Support
  • Answers questions about HoFH and treatment with KYNAMRO
  • Provides encouragement and support for long-term treatment adherence

For more information on
KYNAMRO Cornerstone:

IIPatients are ineligible for the co-pay program if they are eligible to purchase their prescription with benefits from Medicare, including Medicare Part D; Medicaid, including Medicaid Managed Care; Medigap; Veterans Administration (“VA”); Department of Defense (“DoD”); or any similar, federal- or state-funded programs such as pharmaceutical assistance programs. Residents of Massachusetts are prohibited. Void where prohibited by law, taxed, or restricted. Kastle Therapeutics reserves the right to rescind, revoke, or amend these offers without notice.

Contact Us

Call 1-877-KYNAMRO
to learn more and to schedule a visit from your KYNAMRO Clinical Science Specialist.

Email general inquiries to Kastle Therapeutics:

Adverse events should be reported to the Food & Drug Administration (FDA). Visit or call

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References: 1. Cuchel M, Bruckert E, Ginsberg HN, et al; European Atherosclerosis Society Consensus Panel on Familial Hypercholesterolaemia. Homozygous familial hypercholesterolaemia: new insights and guidance for clinicians to improve detection and clinical management. A position paper from the Consensus Panel on Familial Hypercholesterolaemia of the European Atherosclerosis Society. Eur Heart J. 2014;35(32):2146-2157. 2. Raal FJ, Santos RD, Blom DJ, et al. Mipomersen,
an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial. Lancet. 2010;375(9719):998-1006. 3. KYNAMRO [prescribing information]. Chicago, IL; Kastle Therapeutics; 2016. 4. Klose G, Laufs U, März W, Windler E. Familial hypercholesterolemia: developments in diagnosis and treatment. Dtsch Arztebl Int. 2014;111(31-32):523-529. 5. Hopkins PN, Toth PP, Ballantyne CM, Rader DJ; National Lipid Association Expert Panel on Familial Hypercholesterolemia. Familial hypercholesterolemias: prevalence, genetics, diagnosis and screening recommendations from the National Lipid Association Expert Panel on Familial Hypercholesterolemia. J Clin Lipidol. 2011;5(3 suppl):S9-S17. 6. Raal FJ, Santos RD. Homozygous familial hypercholesterolemia: current perspectives on diagnosis and treatment. Atherosclerosis. 2012;223(2):262-268. 7. Data on file. Chicago, IL; Kastle Therapeutics; 2016.