Pronunciation:

et-ran-a-KOE-jeen dez-a-PAR-voe-vek - drlb

Brand Names:

• Hemgenix

Dosage Forms:

• Suspension

Classifications:

Therapeutic—

Antihemophilic Agent

Uses of This Medicine:

Etranacogene dezaparvovec-drlb injection is used to treat hemophilia B (congenital Factor IX deficiency) in patients who are using Factor IX treatment, have current or history of life-threatening bleeding, or have repeated serious bleeding episodes.

This medicine is to be given only by or under the supervision of your doctor and is available only with your doctor's prescription.

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 etranacogene dezaparvovec-drlb injection in children. Safety and efficacy have not been established.

Older adults—

Appropriate studies performed to date have not demonstrated geriatric-specific problems that would limit the usefulness of etranacogene dezaparvovec-drlb injection in the elderly.

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:

• Liver disease (eg, cirrhosis, hepatic fibrosis, hepatitis B or C, non-alcoholic fatty liver disease, chronic alcohol consumption, and non-alcoholic steatohepatitis)—Use with caution. May make these conditions worse.

Proper Use of This Medicine:

A doctor or other trained health professional will give you this medicine. It is given through a needle placed into one of your veins.

Precautions While Using This Medicine:

It is very important that your doctor check you closely and at regular visits to make sure this medicine is working properly. Blood tests will 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. Tell your doctor or nurse right away if you have a rash, itching skin, difficulty with swallowing, dizziness, a fast heartbeat, lightheadedness or fainting, restlessness, trouble breathing, swelling in your face, hands, tongue, or throat, or chest pain after you receive the medicine.

Check with your doctor right away if you have clay-colored stool, dark urine, decreased appetite, fever, itching, loss of appetite, nausea and vomiting, skin rash, stomach pain, swelling of the feet or lower legs, unusual tiredness or weakness, or yellow eyes or skin. These may be symptoms of a serious liver 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

Dizziness

facial swelling

fever or chills

headache

nausea or vomiting

skin rash

trouble breathing

weakness

Less common

Chest discomfort or tightness

fast, pounding, or irregular heartbeat or pulse

hives, itching

irritation

joint pain, stiffness, or swelling

redness or the skin

swelling of the eyelids, face, lips, hands, or feet

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

Cough

diarrhea

general feeling of discomfort or illness

loss of appetite

muscle aches and pains

runny nose

shivering

sore throat

sweating

trouble sleeping

unusual tiredness or weakness

Less common

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.