Fidanacogene elaparvovec

Fidanacogene elaparvovec is used to treat a bleeding disorder called hemophilia B in adult men who are getting preventive treatment for the condition, have ongoing serious bleeding episodes, or have had life-threatening bleeding events in the past.

Your doctor will confirm that you have the right gene type to receive fidanacogene elaparvovec.

Fidanacogene elaparvovec may also be used for other reasons not mentioned in this medication guide.

Your doctor will do tests to ensure fidanacogene elaparvovec is the right treatment for you.

Fidanacogene elaparvovec is given as one dose and is injected into a vein by a healthcare provider.

You will be monitored during and for at least 3 hours after the infusion to ensure you do not have a reaction.

Doses are based on your height and weight. Your dose may change if you gain or lose weight or if you are still growing.

You will need to have your liver function checked once or twice a week for at least 4 months after treatment.

You may be given other medications to help treat abnormal liver function tests. Continue taking these medicines for as long as your doctor prescribes.

Inform your doctor in advance if you need surgery or a medical procedure, or if any bleeding happens after receiving fidanacogene elaparvovec.

Contact your doctor if your symptoms do not improve or if they worsen.

You may need additional medical tests for a longer time after treatment if you have certain conditions or experience certain side effects.

You or your caregiver should practice proper hand hygiene when handling your urine, feces, saliva, mucus, and semen for 6 months after receiving fidanacogene elaparvovec. Place any materials that may have your body waste or fluids in a sealed plastic bag and throw them in regular trash.

fidanacogene elaparvovec may not work for everyone who receives it. Talk to your doctor if you have any questions.

Tell your doctor if you have or have ever had:

• unusual bleeding, any bleeding that will not stop;
• allergy to factor IX complex replacement products;
• hepatitis B or C;
• enlarged spleen;
• HIV (human immunodeficiency virus);
• if you are scheduled to receive a vaccine;
• if you drink large amounts of alcohol; or
• cirrhosis or other liver disease.

Make sure you are up to date on all vaccines before starting treatment with fidanacogene elaparvovec. Your doctor may need to change your vaccination schedule.

Using fidanacogene elaparvovec may increase the risk of developing liver cancer. Ask your doctor about your individual risk.

This medicine is not intended for use by women.

Men using fidanacogene elaparvovec should use condoms or practice abstinence, and avoid donating sperm for up to 6 months after treatment.

Your name should be included on a registry to monitor the effects of this medicine on you.

Get emergency medical help if you notice signs of an allergic reaction: hives, trouble breathing, swelling of your face, lips, tongue, or throat.

Some side effects might happen during or right after the injection. Inform your medical caregiver if you experience dizziness, nausea, light-headedness, itching, sweating, or have a headache, chest tightness, back pain, difficulty breathing, unusual bleeding, or swelling in your face.

Common side effects of fidanacogene elaparvovec may include:

• abnormal liver tests.

This is not a complete list of side effects, and others may occur. Contact your doctor for medical advice about side effects. You can report side effects to the FDA at 1-800-FDA-1088.

Usual Adult Dose for Hemophilia B:

5 x 10(11) vector genomes per kg (vg/kg) of body weight IV once as a single doseCalculation of patient's dose weight:-BMI 30 kg/m2 or less: Dose Weight = Actual body weight-BMI greater than 30 kg/m2: Dose Weight (kg) = 30 kg/m2 x [Height(m)]2Calculation of patient's dose volume in mL: Dose weight in kg divided by 20 = dose in mL-The division factor 20 represents the amount of vector genomes per mL of the suspension (1 x 10[13] vg/mL) divided by the per kg dose (5 x 10[11] vg/kg).Comments:-For one-time single dose IV infusion only-Dosing is based on BMI in kg/m2.-Diluted suspension should be administered as a peripheral IV infusion over approximately 60 minutes (3 mL/min).-Safety and efficacy have not been established in patients 65 years or older.-For examples of dose calculation, refer to the manufacturer product information.Use: For the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who use factor IX prophylaxis therapy, OR have current or historical life-threatening hemorrhage, OR have repeated serious bleeding episodes, AND do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by a US FDA-approved test

Fidanacogene elaparvovec is given as a single dose and does not require a dosing schedule.

In a medical setting, an overdose would be treated quickly.

Do not drink large amounts of alcohol for the first year after treatment. Drinking alcohol may increase your risk of liver damage.

Tell your doctor if you take any medicines for infections, birth control, high cholesterol, seizures, pain, or arthritis (including Advil).

Other drugs may affect fidanacogene elaparvovec, including prescription and over-the-counter medicines, vitamins, and herbal products. Inform your doctor about all other medicines you are using.