Pronunciation:

mi-poe-MER-sen SOE-dee-um

Brand Names:

• Kynamro

Warnings:

Mipomersen sodium can cause elevations in transaminases and also increases hepatic fat (with or without concomitant increases in transaminases). Hepatic steatosis is a risk factor for advanced liver disease, including steatohepatitis and cirrhosis. Measure ALT, AST, alkaline phosphatase, and total bilirubin prior to initiation. Measure ALT and AST regularly. If ALT or AST are 3 times the ULN or greater, interrupt therapy. If clinically significant liver toxicity occurs, discontinue therapy. Mipomersen sodium is available only through a restricted program called the Kynamro(R) REMS. Prescribe mipomersen sodium only to patients with a clinical or laboratory diagnosis consistent with homozygous familial hypercholesterolemia .

Classifications:

Pharmacologic—

Mipomersen

Uses of This Medicine:

Mipomersen injection is used together with a proper diet and other medicines to treat high cholesterol levels in patients with homozygous familial hypercholesterolemia (HoFH).

This medicine is only available through a restricted access program. Only doctors who are enrolled in the restricted program can write a prescription for this medicine.

Before Using This Medicine:

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Children—

Appropriate studies have not been performed on the relationship of age to the effects of mipomersen injection in the pediatric population. Safety and efficacy have not been established.

Older adults—

In clinical studies, elderly patients receiving mipomersen had higher rates of high blood pressure and swelling compared with other elderly patients not receiving this medication and younger patients receiving the medication. Elderly patients receiving mipomersen were also more likely to develop fatty liver than younger patients.

Breast-feeding—

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Other medicines—

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Other interactions—

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Other medical problems—

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

• Kidney disease, severe or
• Patients receiving dialysis or
• Proteinuria (protein in the urine)—Use is not recommended in patients with these conditions.

• Liver disease, active or
• Liver disease, moderate or severe or
• Liver enzymes, persistently elevated—Should not be used in patients with these conditions.

Proper Use of This Medicine:

This medicine is given as a shot under your skin, usually in the abdomen or stomach, thighs, or upper arms.

Mipomersen may sometimes be given at home to patients who do not need to be in the hospital. If you are using this medicine at home, your doctor will teach you how to prepare and inject the medicine. Be sure that you understand exactly how the medicine is prepared and injected.

You will be shown the body areas where this shot can be given. Use a different body area each time you give yourself a shot. Keep track of where you give each shot to make sure you rotate body areas. This will help prevent skin problems from the injections.

This medicine is usually given once a week, on the same day every week. Do not inject on skin areas that have cuts, scrapes, infection, scars, sunburns, or tattoos.

The prefilled syringe should be removed from the refrigerator and allowed to reach room temperature for at least 30 minutes prior to injection. Do not remove the needle cover of the prefilled syringe while allowing the syringe to reach room temperature.

Use each syringe only one time. Do not save an open syringe. If the medicine in the syringe has changed color, or if you see particles in it, do not use it.

In addition to this medicine, your doctor may change your diet to one that is low in fat, sugar, and cholesterol. Carefully follow your doctor's order about any special diet.

This medicine should come with a Medication Guide. Read and follow these instructions carefully. Ask your doctor or pharmacist if you have any questions.

Dosing—

The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.

The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.

• For injection dosage form:
  • For high cholesterol:
    • Adults—200 milligrams (mg) injected under your skin once a week.
    • Children—Use and dose must be determined by your doctor.

Missed dose—

This medicine needs to be given on a fixed schedule. If you miss a dose or forget to use your medicine, call your doctor or pharmacist for instructions.

If you missed a dose of this medicine, use this medicine at least 3 days before the next weekly dose.

Storage—

Keep out of the reach of children.

Do not keep outdated medicine or medicine no longer needed.

Ask your healthcare professional how you should dispose of any medicine you do not use.

Store in the refrigerator. Do not freeze.

You may also store the medicine at or below 30 degrees C (86 degrees F), away from heat sources, for up to 14 days. Protect the medicine from light. Keep your medicine in the original package until you are ready to use it.

Throw away used needles in a hard, closed container that the needles cannot poke through. Keep this container away from children and pets.

Precautions While Using This Medicine:

It is very important that your doctor check your progress at regular visits. This will allow your doctor to see if the medicine is working properly to lower your cholesterol levels and to decide if you should continue to use it. Blood tests may be needed to check for unwanted effects.

Check with your doctor right away if you have pain or tenderness in the upper stomach, pale stools, dark urine, loss of appetite, nausea, vomiting, or yellow eyes or skin. These could be symptoms of a liver injury.

Tell your doctor if you have redness, pain, tenderness, itching, or swelling at the injection site. Also, tell your doctor if you have flu-like symptoms (eg, fever, chills, joint or muscle pain, unusual tiredness or weakness) after receiving this medicine.

Drinking alcohol while using this medicine may increase your risk of liver injury. It is recommended that you drink no more than one alcoholic drink per day while using this medicine.

Make sure your doctor knows if you are pregnant or planning to become pregnant. You must use an effective form of birth control to keep from getting pregnant. Talk to your doctor about effective birth control.

Do not take other medicines unless they have been discussed with your doctor. This includes prescription or nonprescription (over-the-counter [OTC]) medicines and herbal or vitamin supplements.

Side Effects of This Medicine:

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur:

More common

Chills

cough

dark urine

diarrhea

fever

general feeling of discomfort or illness

headache

joint pain

light-colored stools

loss of appetite

muscle aches and pains

nausea and vomiting

runny nose

shivering

sore throat

sweating

trouble sleeping

unusual tiredness or weakness

upper right abdominal or stomach pain

vomiting

yellow eyes and skin

Less common

Arm, back, or jaw pain

bloating or swelling of the face, arms, hands, lower legs, or feet

chest pain or discomfort

chest tightness or heaviness

fast or irregular heartbeat

irregular, pounding, or racing heartbeat or pulse

rapid weight gain

shortness of breath

sweating

tingling of the hands or feet

unusual weight gain or loss

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

Bleeding, blistering, burning, coldness, discoloration of the skin, feeling of pressure, hives, infection, inflammation, itching, lumps, numbness, pain, rash, redness, scarring, soreness, stinging, swelling, tenderness, tingling, ulceration, or warmth at the injection site

pain in the arms or legs

Less common

Abdominal or stomach pain

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.