Valoctocogene roxaparvovec: Patient drug information

What is this drug used for?

What do I need to tell my doctor BEFORE I take this drug?

If you are allergic to this drug; any part of this drug; or any other drugs, foods, or substances. Tell your doctor about the allergy and what signs you had.
If you have had a positive test for factor VIII inhibitor or antibodies to AAV5.
If you have an infection.
If you have liver disease.
If you are taking efavirenz or isotretinoin.
This is not a list of all drugs or health problems that interact with this drug.
Tell your doctor and pharmacist about all of your drugs (prescription or OTC, natural products, vitamins) and health problems. You must check to make sure that it is safe for you to take this drug with all of your drugs and health problems. Do not start, stop, or change the dose of any drug without checking with your doctor.

What are some things I need to know or do while I take this drug?

Tell all of your health care providers that you take this drug. This includes your doctors, nurses, pharmacists, and dentists.
Have your blood work and other lab tests checked as you have been told by your doctor.
Do not drink alcohol for at least 1 year after taking this drug. You may also need to limit alcohol after that time. Talk with your doctor.
Make sure you are up to date with all your vaccines before treatment with this drug.
Talk with your doctor before getting any vaccines. Use of some vaccines with this drug may either raise the chance of an infection or make the vaccine not work as well.
There is a chance that this drug may cause liver cancer. No cases of cancer from this drug have been reported. Tell your doctor if you have a risk for liver cancer, or if you have any questions about this information.
Do not donate blood, organs, tissues, or cells after getting this drug.
This drug is found in semen. Do not donate semen for 6 months after you take this drug.
If your sex partner may get pregnant, you must use birth control for 6 months after you take this drug. If your partner gets pregnant, call the doctor right away.
Tell your doctor if you are pregnant, plan on getting pregnant, or are breast-feeding. You will need to talk about the benefits and risks to you and the baby. This drug is not approved for use in women.

What are some side effects that I need to call my doctor about right away?

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:
Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.
Signs of liver problems like dark urine, tiredness, decreased appetite, upset stomach or stomach pain, light-colored stools, throwing up, or yellow skin or eyes.
Call your doctor right away if you have signs of a blood clot like chest, arm, back, neck, or jaw pain or pressure; coughing up blood; numbness or weakness on 1 side of your body; trouble speaking or thinking; change in balance; change in eyesight; shortness of breath; or swelling, warmth, or pain in the leg or arm.
Infusion reactions have happened with this drug. Tell your doctor right away if you have fever, chills, rash, hives, itching, sneezing, coughing, shortness of breath, runny or stuffy nose, watery eyes, tingling throat, upset stomach, diarrhea, dizziness, passing out, or fast heartbeat. You may be watched for a reaction for some time after taking this drug.

What are some other side effects of this drug?

All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your doctor or get medical help if any of these side effects or any other side effects bother you or do not go away:
Upset stomach or throwing up.
Stomach pain.
Headache.
Feeling tired or weak.
These are not all of the side effects that may occur. If you have questions about side effects, call your doctor. Call your doctor for medical advice about side effects.
You may report side effects to your national health agency.

How is this drug best taken?

Use this drug as ordered by your doctor. Read all information given to you. Follow all instructions closely.
It is given as an infusion into a vein over a period of time.

What do I do if I miss a dose?

How do I store and/or throw out this drug?

If you need to store this drug at home, talk with your doctor, nurse, or pharmacist about how to store it.

General drug facts

If your symptoms or health problems do not get better or if they become worse, call your doctor.
Do not share your drugs with others and do not take anyone else's drugs.
Keep all drugs in a safe place. Keep all drugs out of the reach of children and pets.
Throw away unused or expired drugs. Do not flush down a toilet or pour down a drain unless you are told to do so. Check with your pharmacist if you have questions about the best way to throw out drugs. There may be drug take-back programs in your area.
Some drugs may have another patient information leaflet. If you have any questions about this drug, please talk with your doctor, nurse, pharmacist, or other health care provider.
If you think there has been an overdose, call your poison control center or get medical care right away. Be ready to tell or show what was taken, how much, and when it happened.

Consumer Information Use and Disclaimer

This generalized information is a limited summary of diagnosis, treatment, and/or medication information. It is not meant to be comprehensive and should be used as a tool to help the user understand and/or assess potential diagnostic and treatment options. It does NOT include all information about conditions, treatments, medications, side effects, or risks that may apply to a specific patient. It is not intended to be medical advice or a substitute for the medical advice, diagnosis, or treatment of a health care provider based on the health care provider's examination and assessment of a patient's specific and unique circumstances. Patients must speak with a health care provider for complete information about their health, medical questions, and treatment options, including any risks or benefits regarding use of medications. This information does not endorse any treatments or medications as safe, effective, or approved for treating a specific patient. UpToDate, Inc. and its affiliates disclaim any warranty or liability relating to this information or the use thereof. The use of this information is governed by the Terms of Use, available at https://www.wolterskluwer.com/en/know/clinical-effectiveness-terms.