Etranacogene dezaparvovec-drlb (Intravenous)

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

This medicine should only be given by your doctor or under their supervision and is available only with your doctor's prescription.

It is very important that your doctor checks you closely and regularly to ensure 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 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.

In deciding to use a medicine, you and your doctor need to consider the risks of taking it against its benefits. For this medicine, please keep the following in mind:

Allergies

Inform your doctor if you have ever had an unusual or allergic reaction to this medicine or any other medicines. Also, let your healthcare professional know if you have any other allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, carefully read the label or package ingredients.

Pediatric

Studies have not been done to assess how age affects the use of etranacogene dezaparvovec-drlb injection in children. Its safety and effectiveness have not been confirmed for this age group.

Geriatric

Current studies have not shown any specific problems in elderly patients that would limit the usefulness of etranacogene dezaparvovec-drlb injection.

Breast Feeding

There are no sufficient studies in women to determine the risk to infants when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Interactions with Medicines

Some medicines should not be used together at all, while in other cases, two different medicines may be taken at the same time even if an interaction could happen. In these situations, your doctor may adjust the dose or take other precautions. Inform your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Interactions with Food/Tobacco/Alcohol

Some medicines should not be taken around the time of eating food or certain types of food due to potential interactions. Using alcohol or tobacco with some medicines may also lead to interactions. Talk with your healthcare professional about the use of your medicine with food, alcohol, or tobacco.

Other Medical Problems

Having other medical issues may affect how you can use this medicine. Be sure to tell your doctor if you have any other medical problems, especially:

• Liver disease (e.g., cirrhosis, hepatic fibrosis, hepatitis B or C, non-alcoholic fatty liver disease, chronic alcohol consumption, and non-alcoholic steatohepatitis)—Use with caution. This may worsen these conditions.

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

In the U.S.

• Hemgenix

Available Dosage Forms:

• Suspension

Therapeutic Class: Antihemophilic Agent

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

• Why is Hemgenix so expensive?