Valoctocogene roxaparvovec

Valoctocogene roxaparvovec is used to treat severe hemophilia A in adults.

Your doctor will ensure that you can be treated with valoctocogene roxaparvovec.

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

Valoctocogene roxaparvovec may also be used for reasons not mentioned in this medication guide.

Your doctor will do medical tests before starting treatment with valoctocogene roxaparvovec to ensure it is the right treatment for you.

Valoctocogene roxaparvovec is given as a single dose through an infusion into a vein. A healthcare provider will give you this injection.

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

You will need weekly blood tests to check your liver function for at least 26 weeks after treatment. You may need other medical tests for a longer time if you have certain medical conditions or side effects.

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

Inform your doctor in advance if you need surgery or a medical procedure, or if any bleeding occurs after receiving valoctocogene roxaparvovec. You may need to start another medicine to treat hemophilia A. Follow your doctor's instructions.

Tell your doctor if you are using other medicines or have other medical conditions or allergies.

You should not be treated with valoctocogene roxaparvovec if you have:

• any active, or uncontrolled chronic infection;
• an allergy to mannitol; or
• cirrhosis or stage 3 or 4 liver fibrosis.

Tell your doctor if you have or have ever had:

• heart disease, a blood clot or stroke;
• any type of hepatitis, HIV;
• a planned surgery or medical procedure;
• abnormal liver function tests;
• if you drink alcohol; or
• any other allergies.

Using valoctocogene roxaparvovec may increase your risk of developing certain types of cancer including liver cancer. Ask your doctor about your individual risk.

Tell your doctor if you recently received a vaccine or are scheduled to receive a vaccine. Your doctor may need to adjust your vaccination schedule.

Make sure you are current on all vaccines before you start using valoctocogene roxaparvovec.

Your name should be listed on a registry to track the effects of valoctocogene roxaparvovec on you.

This medicine is not intended for use by women.

Men and their female partners should use effective birth control to prevent pregnancy. If you are a man, use birth control if your sex partner is able to get pregnant. Keep using birth control for at least 6 months after your last dose. Ask your doctor about birth control options. Tell your doctor right away if a pregnancy occurs.

Men using valoctocogene roxaparvovec should not donate semen for at least 6 months after treatment.

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

Some side effects may happen during or right after the injection. Let your medical caregiver know if you feel dizzy, nauseous, light-headed, itchy, sweaty, or have a headache, chest tightness, back pain, difficulty breathing, unusual bleeding, or swelling in your face.

Also, inform your doctor immediately if you experience:

• liver problems, which may include loss of appetite, nausea, vomiting, stomach pain (upper right side), tiredness, itching, dark urine, clay-colored stools, or jaundice (yellowing of the skin or eyes); or
• signs of a blood clot, such as sudden numbness or weakness (especially on one side of the body), severe headache, slurred speech, chest pain, shortness of breath, dizziness, coughing up blood, or pain, swelling, or warmth in one leg.

Common side effects of valoctocogene roxaparvovec may include:

• nausea, vomiting, stomach pain;
• abnormal blood tests;
• a reaction after receiving the injection; or
• feeling weak or tired, headache.

This is not a complete list of side effects, and others may occur. Call 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 A:

6 × 10(13) vg/kg as a single IV infusion at a rate of 1 mL/minDuration of therapy: OnceComments:-Do not administer this drug to patients with a positive test for antibodies to AAV5, factor VIII inhibitor, active acute or uncontrolled chronic infections, known significant hepatic fibrosis or cirrhosis, or mannitol hypersensitivity.-To determine dose needed in mL, multiply patient body weight in kg by 3.-To determine number of vials to be thawed, divide the patient dose volume (mL) by 8.-Infuse the suspension through a suitable peripheral vein using an infusion catheter with an in-line filter and a programmable syringe pump.-If tolerated, the rate of infusion may be increased every 30 minutes by 1 mL/min up to a maximum rate of 4 mL/min.-Treatment with this drug should be carried out under the supervision of a physician experienced in hemophilia/bleeding disorder treatment.-Safety and effectiveness of this drug in patients with prior or active factor VIII inhibitors have not been established.Use: For the treatment of adults with severe hemophilia A without pre-existing antibodies to adeno-associated virus serotype 5

Valoctocogene roxaparvovec is given in one dose. Call your doctor for instructions if you miss an appointment for your valoctocogene roxaparvovec injection.

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

Ask your doctor before using any other medicine for hemophilia A.

Do not drink alcohol for at least a year after receiving this treatment. Dangerous side effects could occur.

Avoid receiving a "live" vaccine while you are being treated with valoctocogene roxaparvovec.

Do not donate blood, organs, tissues, or cells for transplantation after receiving valoctocogene roxaparvovec.

Valoctocogene roxaparvovec can cause liver damage, especially if you take certain health supplements, steroids, or other medications for infections, birth control, high cholesterol, seizures, pain, or arthritis (including Advil).

Inform your doctor about all your other medications, especially:

• isotretinoin; or
• efavirenz.

Other drugs may impact valoctocogene roxaparvovec, including both prescription and over-the-counter medicines, vitamins, and herbal products. Be sure to tell your doctor about all other medications you are using.