Pronunciation:

goe-loe-DIR-sen

Brand Names:

• VYONDYS 53

Dosage Forms:

• Solution

Classifications:

Therapeutic—

Musculoskeletal Agent

Pharmacologic—

Antisense Oligonucleotide

Uses of This Medicine:

Golodirsen 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 golodirsen injection in children.

Older adults—

No information is available on the relationship of age to the effects of golodirsen 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. May make this condition worse.

Proper Use of This Medicine:

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

Precautions While Using This Medicine:

Your doctor will check your or your child's progress closely while 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.

This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Check with your doctor right away if you have chest tightness, cough, difficulty swallowing, dizziness, fast heartbeat, hives, itching, skin rash, puffiness or swelling around the eyes, face, lips, or tongue, trouble breathing, or unusual tiredness or weakness.

Check with your doctor right away if you or your child have 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

Fast, pounding, or irregular heartbeat or pulse

fever

Incidence not known

Agitation

chest tightness

coma

confusion

cough

decreased urine output

depression

difficulty swallowing

dizziness

fast heartbeat

headache

hives, itching, skin rash

hostility

irritability

lethargy

muscle twitching

nausea

puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue

rapid weight gain

seizures

stupor

swelling of the face, ankles, or hands

trouble breathing

unusual tiredness or weakness

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

Back pain

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

constipation

diarrhea

falling

headache

muscle aches

sore throat

stomach pain

stuffy or runny nose

vomiting

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.