Fidanacogene elaparvovec-dzkt Injection

WHY is this medicine prescribed?

Fidanacogene elaparvovec-dzkt injection is used to treat moderate to severe 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. Fidanacogene dezaparvovec-dzkt is in a class of medications called gene therapy. It works by helping your body to increase blood clotting factor IX availability, 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?

Fidanacogene dezaparvovec-dzkt 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 60 minutes as a single dose.

Fidanacogene dezaparvovec-dzkt 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, nausea, vomiting, 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 fidanacogene elaparvovec-dzkt injection,

• tell your doctor and pharmacist if you are allergic to fidanacogene elaparvovec-dzkt, blood clotting factor IX products, any other medications, or any of the ingredients in fidanacogene elaparvovec-dzkt 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 human immunodeficiency virus (HIV) or 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 fidanacogene elaparvovec-dzkt injection is intended only for use in adult men. Fidanacogene elaparvovec-dzkt is not intended for use in women. If you are male and your partner can become pregnant, you should use a condom for 6 months after you receive fidanacogene elaparvovec-dzkt injection. It is not known if fidanacogene elaparvovec-dzkt injection may harm the baby if it is used by women who are pregnant, may become pregnant, or are breastfeeding.

• ask your doctor about the safe use of alcoholic beverages after you receive fidanacogene elaparvovec-dzkt injection. Alcohol may decrease the effect of this medication and can make the side effects from fidanacogene elaparvovec-dzkt injection worse.

• do not donate blood, organs, tissues, sperm, or cells for transplantation after you receive fidanacogene elaparvovec-dzkt injection.

• check with your doctor to see if you need to receive any vaccinations. Do not have any vaccinations without talking to your doctor.

• you should know that fidanacogene elaparvovec-dzkt 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 fidanacogene elaparvovec-dzkt 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.

• you should know that fidanacogene elaparvovec-dzkt injection contains a virus that could spread and infect other people. You should maintain proper hygiene and wash your hands if you come in contact with your bodily fluids or waste (urine, feces, saliva). You should be sure to put all contaminated materials that may contain your bodily fluids or waste into a sealed plastic bag and throw them away into the garbage for 6 months after the infusion.

What SPECIAL DIETARY instructions should I follow?

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

What SIDE EFFECTS can this medicine cause?

Fidanacogene elaparvovec-dzkt injection may cause side effects. 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

Fidanacogene elaparvovec-dzkt injection may increase your risk of developing hepatocellular cancer (a type of liver cancer) or other types of cancer. Your doctor may order tests to monitor your liver for 5 years after treatment with fidanacogene elaparvovec-dzkt injection. Talk to your doctor about the risks of receiving this medication.

Fidanacogene elaparvovec-dzkt 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 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 fidanacogene elaparvovec-dzkt injection.

Fidanacogene elaparvovec-dzkt injection may only be used in certain adults with hemophilia B. Your doctor will order a blood test to help decide if this medication is right for you.

Before having any laboratory test, tell your doctor and the laboratory personnel that you have received fidanacogene elaparvovec-dzkt 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.