Pronunciation:

kas-i-MER-sen

Brand Names:

• Amondys 45

Dosage Forms:

• Solution

Classifications:

Therapeutic—

Musculoskeletal Agent

Pharmacologic—

Antisense Oligonucleotide

Uses of This Medicine:

Casimersen injection is used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed specific genetic mutation. DMD is a rare, inherited muscle disease that is caused by absence of a protein called dystrophin, which helps keep muscle cells intact.

This medicine is to be given only by or under the direct supervision of a doctor.

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 performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of casimersen injection in children.

Older adults—

No information is available on the relationship of age to the effects of casimersen injection in geriatric patients. DMD is usually a disease of children and young adults.

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—Use with caution. The effects may be increased because of slower removal of the medicine from the body.

Proper Use of This Medicine:

A nurse or other trained health professional will give you or your child this medicine in a medical facility. This medicine is given through a needle placed in one of your veins. It must be given slowly, so the needle should remain in place for 35 to 60 minutes once a week.

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.

Precautions While Using This Medicine:

Your doctor will check your or your child's progress closely while you are receiving this medicine to see if it is working properly and to decide if you or your child should continue to receive it. Blood and urine tests may be needed to check for unwanted effects.

Check with your doctor right away if you or your child has blood in the urine, change in frequency of urination or amount of urine, drowsiness, increased thirst, loss of appetite, nausea or vomiting, swelling of the feet or lower legs, trouble breathing, or weakness. These could be symptoms of a serious kidney problem.

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 or nurse immediately if any of the following side effects occur:

More common

Body aches or pain

chills

cough

difficulty in moving

dizziness

ear congestion

fast, pounding, or irregular heartbeat

fever

headache

joint pain

lightheadedness

loss of voice

muscle pain or stiffness

nausea

runny or stuffy nose

sneezing

sore throat

stomach pain

swelling of the face, ankles, or hands

tender, swollen glands in the neck

trouble breathing

trouble swallowing

unusual tiredness or weakness

vomiting

Incidence not known

Agitation

blood in the urine

coma

confusion

decreased urine output

depression

irritability

lethargy

muscle twitching

rapid weight gain

seizures

stupor

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 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

change in hearing

earache or pain in the ear

ear drainage

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.