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Overview: Roctavian is used to treat hemophilia A. It works by helping your liver produce factor VIII. Common side effects include nausea and vomiting, tiredness, headache, and stomach pain. Serious side effects include infusion reactions, liver damage, and blood clots. Your health care provider will give you Roctavian as a single, one-time infusion into a vein.

Uses

What is Roctavian used for?

Roctavian (valoctocogene roxaparvovec-rvox) is used for certain people with an inherited condition called hemophilia A, also known as congenital factor VIII deficiency. 

Your health care provider can do a test to see if you are able to receive Roctavian.

Roctavian may also be used for other conditions as determined by your health care provider.

How does Roctavian work (mechanism of action)?

People with hemophilia A have low levels of an important blood protein called factor VIII. Roctavian works by helping your liver produce factor VIII in the body.

How is Roctavian supplied (dosage forms)?

Roctavian is available in an injectable form that is given by a health care provider.

How should I store Roctavian?

Roctavian is usually given by a health care provider in a hospital or clinic. You will not store it at home.

Side Effects

What are the most common side effects of Roctavian?

The most common side effects of Roctavian are listed below. Tell your health care provider if you have any of these side effects that bother you.

  • Nausea
  • Tiredness
  • Headache
  • Infusion reactions (see below)
  • Vomiting
  • Stomach pain

There may be other side effects of Roctavian that are not listed here. Contact your health care provider if you think you are having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088 (800-332-1088). In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.

What are the serious side effects of Roctavian?

While less common, the most serious side effects of Roctavian are described below, along with what to do if they happen.

Severe Allergic Reactions. Roctavian may cause allergic reactions, which can be serious. Get help right away if you have any of the following symptoms of a serious allergic reaction.

  • Breathing problems or wheezing
  • Racing heart
  • Fever or general ill feeling
  • Swollen lymph nodes
  • Swelling of the face, lips, mouth, tongue, or throat
  • Trouble swallowing or throat tightness
  • Itching or skin rash
  • Bumps on the skin called hives that can be red, pink, white, or brown depending on your skin tone
  • Nausea or vomiting
  • Dizziness, feeling lightheaded, or fainting
  • Stomach cramps
  • Joint pain

Infusion Reactions. Roctavian can cause reactions that can be serious when it is given to you. Your infusion may need to be slowed or stopped if you have these reactions. Tell your health care provider right away if you have any of the following symptoms during and after your infusion.

  • Hives, itching, or skin rash
  • Sneezing, coughing, runny nose, or watery eyes
  • Shortness of breath
  • Tingling throat
  • Nausea
  • Diarrhea
  • Low blood pressure
  • Fast heartbeat
  • Feeling faint
  • Fever, shivering, or chills

Liver Damage. Liver damage, also called hepatotoxicity, can happen with Roctavian. Keep all appointments to check the health of your liver. Call your health care provider right away if you have any of the following symptoms of liver damage.

  • Nausea or vomiting
  • Stomach or belly pain
  • Fever
  • Weakness or unusual tiredness
  • Itching
  • Loss of appetite
  • Light-colored poop
  • Dark-colored urine (pee)
  • Your skin or the whites of your eyes turning yellowish in color (also called jaundice)

Blood Clots. Roctavian may increase the risk of a blood clot in your arteries or veins, which could cause a stroke, heart attack, or venous thromboembolism, including a blood clot in the lungs (pulmonary embolism) or deep vein thrombosis (DVT). The risk may be higher if you are already at risk of a blood clot. Get emergency help if you have any of the following signs of blood clots. 

  • Chest pain or pressure
  • Fast or abnormal heartbeat
  • Trouble breathing
  • Pain in your back, jaw, throat, or arm
  • Sweating, upset stomach, vomiting, or dizziness
  • Sudden weakness on one side of your body
  • Sudden and severe headache
  • Slurred speech or trouble understanding speech
  • Pain, swelling, or tenderness in your arm or leg
  • Vision loss
  • Bulging eyes 
  • Double vision

Cancer. Roctavian might cause a new cancer to develop or cause a previously treated cancer to recur. Tell your health care provider about any risk factors you have for liver cancer, such as hepatitis B, hepatitis C, or fatty liver disease. Keep all monitoring appointments with your health care provider and tell your health care provider about any new symptoms that you may get.

Warnings & Precautions

Who should not use Roctavian?

Allergies to Ingredients. People who are allergic to any of the following should not use Roctavian.

  • Mannitol
  • Roctavian
  • Valoctocogene roxaparvovec-rvox
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in Roctavian.

Infection. Roctavian should not be used if you have an active infection (such as a respiratory infection), or if you have an infection for a long time that is not controlled by the medicines you take (such as chronic active hepatitis B).

Liver Scarring. Roctavian should not be used if you have significant scarring of your liver (hepatic fibrosis) or cirrhosis.

What should I know about Roctavian before using it?

Roctavian is usually given by a health care provider in a hospital or clinic. 

Your health care provider will give you Roctavian as a single, one-time infusion injected into a vein.

Keep all appointments with your health care provider, including for blood work and other tests, before and after receiving Roctavian. Tell your health care provider if you start bleeding after receiving Roctavian.

You may need to take certain medicines after receiving Roctavian. Follow your health care provider's instructions and let them know if you have any questions.

You and any partner who can become pregnant must avoid becoming pregnant for 6 months after receiving Roctavian. If you have concerns or have questions about your birth control options, talk to your health care provider.

Do not donate semen for at least 6 months after receiving Roctavian.

Do not donate blood, organs, tissues, or cells for transplants after receiving Roctavian.

What should I tell my health care provider before using Roctavian?

Tell your health care provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them know if Roctavian is right for you.

In particular, make sure that you discuss any of the following.

Current and Past Health Conditions. Tell your health care provider if you have any of the following.

Other Medicines and Supplements. Roctavian may interact with other medicines and supplements. Before receiving Roctavian, tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. See the Interactions section for more details.

Vaccines. Talk with your health care provider about any vaccines that you may need. You may not be able to receive certain vaccines after receiving Roctavian. Tell your health care provider about any vaccines you have recently received or have scheduled.

Pregnancy. People who can become pregnant are not usually prescribed Roctavian.

Breastfeeding. People who can breastfeed are not usually prescribed Roctavian.

How can I get more information about Roctavian?

For more information about Roctavian, you can visit the drugmaker’s website at www.roctavian.com or call them at 866-906-6100.

Interactions

Does Roctavian interact with foods or drinks?

There are no known interactions between Roctavian and foods or drinks.

Avoid drinking alcohol for at least one year after receiving Roctavian. After that, talk with your health care provider about how much alcohol may be acceptable. The risk of liver problems may be increased if you drink alcohol after receiving Roctavian.

Does Roctavian interact with other medicines (drug interactions)?

Always tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using.

In particular, make sure that you discuss if you are using any of the following before using Roctavian.

  • A medicine for HIV or AIDS
  • Isotretinoin, which is a medicine used to treat acne

This may not be a complete list of medicines that can interact with Roctavian. Always check with your health care provider.

Does interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose/Missed Dose

What should I do if I accidentally use too much Roctavian?

If you or someone else has used too much Roctavian, get medical help right away, call 911, or contact a Poison Control center at 800-222-1222.

What should I do if I miss a dose of Roctavian?

Roctavian is usually given by a health care provider in a hospital or clinic. If you miss an appointment to get your Roctavian dose, call to make another appointment as soon as possible.