Etranacogene dezaparvovec-drlb Injection

WHY is this medicine prescribed?

Etranacogene dezaparvovec-drlb injection is used to treat hemophilia B (an inherited condition in which the blood does not clot normally because of a missing blood clotting factor IX) in certain adults who are receiving factor IX replacement therapy to prevent bleeding or who have had or are having life-threatening bleeding or who are having repeated, spontaneous bleeding episodes. Etranacogene dezaparvovec-drlb is in a class of medications called gene therapy. It works by helping your body to increase blood clotting factor IX available which may help stop bleeding.

Are there OTHER USES for this medicine?

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

HOW should this medicine be used?

Etranacogene dezaparvovec-drlb comes as a suspension (liquid) to be injected intravenously (into a vein) by a doctor or nurse in a healthcare setting. It is usually infused slowly over 1 to 2 hours as a single dose.

Etranacogene dezaparvovec-drlb may cause serious reactions during or after you receive your infusion. If you experience any of the following symptoms, tell your doctor immediately: flushing, fever, chills, flu-like symptoms, chest pain, headache, stomach pain, dizziness, feeling faint, shortness of breath, rash or hives, difficulty breathing, or swelling of the face or throat. A doctor or nurse will watch you carefully for these side effects while the medication is being infused, and for at least 3 hours after. Your doctor may need to slow down or temporarily stop your infusion if you experience these or other side effects.

Ask your pharmacist or doctor for a copy of the manufacturer's information for the patient.

What SPECIAL PRECAUTIONS should I follow?

Before receiving etranacogene dezaparvovec-drlb injection,

• tell your doctor and pharmacist if you are allergic to etranacogene dezaparvovec-drlb, any other medications, or any of the ingredients in etranacogene dezaparvovec-drlb injection. Ask your pharmacist for a list of the ingredients.

• tell your doctor and pharmacist what other prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking or plan to take. Your doctor may need to change the doses of your medications or monitor you carefully for side effects.

• tell your doctor if you regularly drink alcohol or sometimes drink large amounts of alcohol, or if you have or have ever had any liver problems, including hepatitis B or C (a virus that infects the liver and may cause severe liver damage) or cirrhosis (scarring) of the liver.

• you should know that etranacogene dezaparvovec-drlb injection is intended only for use in adult men. Entranacogene dezaparvovec-drlb is not intended for use in women. It is not known if etranacogene dezaparvovec-drlb injection may harm the baby if it is used by women who are pregnant, may become pregnant, or are breastfeeding.

• do not donate blood, organs, tissues, sperm, or cells for transplantation after you receive etranacogene dezaparvovec-drlb injection.

• you should know that etranacogene dezaparvovec-drlb injection may cause liver damage. Your doctor may tell you to have an ultrasound (a type of imaging to look at organs and structures inside the body) of your liver before and after your treatment with etranacogene dezaparvovec-drlb injection. If you experience any of the following symptoms, call your doctor immediately: extreme tiredness, pain in the upper right part of the stomach, nausea, yellowing of the skin or eyes, dark yellow urine, or pale stools.

What SPECIAL DIETARY instructions should I follow?

Unless your doctor tells you otherwise, continue your normal diet.

What SIDE EFFECTS can this medicine cause?

Etranacogene dezaparvovec-drlb injection may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:

• headache
• nausea
• tiredness
• feeling unwell

Some side effects can be serious. If you experience any of these symptoms or those listed in the SPECIAL PRECAUTIONS section, call your doctor immediately or get emergency medical treatment:

• unusual bleeding or bruising

Etranacogene dezaparvovec-drlb injection may increase your risk of developing hepatocellular cancer (a type of liver cancer). Talk to your doctor about the risks of receiving this medication.

Etranacogene dezaparvovec-drlb injection may cause other side effects. Call your doctor if you have any unusual problems while taking this medication.

If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration's (FDA) MedWatch Adverse Event Reporting program online (http://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).

What should I do in case of OVERDOSE?

In case of overdose, call the poison control helpline at 1-800-222-1222. Information is also available online at https://www.poisonhelp.org/help. If the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services at 911.

What OTHER INFORMATION should I know?

Keep all appointments with your doctor and the laboratory. Your doctor will order certain lab tests before and after your treatment to check your body's response to etranacogene dezaparvovec-drlb injection.

Before having any laboratory test, tell your doctor and the laboratory personnel that you have received etranacogene dezaparvovec-drlb injection.

It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.