Pronunciation:

val-ok-toe-KOE-jeen rox-a-PAR-voe-vek - rvox

Brand Names:

• Roctavian

Dosage Forms:

• Suspension

Classifications:

Therapeutic—

Antihemophilic Agent

Uses of This Medicine:

Valoctocogene roxaparvovec-rvox injection is used to treat severe hemophilia A in patients who do not have antibodies to adeno-associated virus serotype 5 (AAV5).

This medicine is to be given only by or under the supervision of your doctor.

Before Using This Medicine:

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Children—

Appropriate studies have not been performed on the relationship of age to the effects of valoctocogene roxaparvovec-rvox injection in the pediatric population. Safety and efficacy have not been established.

Older adults—

Appropriate studies performed to date have not demonstrated geriatric-specific problems that would limit the usefulness of valoctocogene roxaparvovec-rvox injection in the elderly.

Breast-feeding—

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Other medicines—

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. When you are receiving this medicine, it is especially important that your healthcare professional know if you are taking any of the medicines listed below. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.

Using this medicine with any of the following medicines is usually not recommended, but may be required in some cases. If both medicines are prescribed together, your doctor may change the dose or how often you use one or both of the medicines.

• Adenovirus Vaccine, Live
• Bacillus of Calmette and Guerin Vaccine, Live
• Cholera Vaccine, Live
• Dengue Tetravalent Vaccine, Live
• Ebola Zaire Vaccine, Live
• Efavirenz
• Influenza Virus Vaccine, Live
• Isotretinoin
• Measles Virus Vaccine, Live
• Mumps Virus Vaccine, Live
• Poliovirus Vaccine, Live
• Rotavirus Vaccine, Live
• Rubella Virus Vaccine, Live
• Smallpox Monkeypox Vaccine, Live Non-Replicating
• Smallpox Vaccine
• Typhoid Vaccine, Live
• Varicella Virus Vaccine, Live
• Yellow Fever Vaccine
• Zoster Vaccine, Live

Other interactions—

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Other medical problems—

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

• Allergy to mannitol or
• Infection, active or uncontrolled or
• Liver disease (eg, liver fibrosis or cirrhosis)—Should not be use in patients with these conditions.

• Blood clots or
• Cancer or
• Heart or blood vessel disease or
• Infection or
• Liver disease (eg, hepatitis B or C, non-alcoholic fatty liver disease, chronic alcohol consumption, and non-alcoholic steatohepatitis)—Use with caution. May make these conditions worse.

Proper Use of This Medicine:

A doctor or other trained health professional will give you this medicine. It is given through a needle placed into one of your veins. It must be given slowly, so the needle will have to remain in place for at least 2 hours.

Precautions While Using This Medicine:

It is very important that your doctor check you closely and at regular visits to make sure this medicine is working properly. Blood tests will be needed to check for unwanted effects.

Do not drink alcohol during treatment and for at least 1 year after receiving this medicine.

Women should not use this medicine, especially women who are pregnant or able to become pregnant. This medicine can harm an unborn baby. It can also cause birth defects if the father is using it when his sexual partner becomes pregnant. Males with female partners who can become pregnant must use an effective birth control during treatment and for at least 6 months after the last dose. If a pregnancy occurs while you are using this medicine, tell your doctor right away.

This medicine may cause infusion-related reactions. Check with your doctor right away if you have back pain, chest tightness, chills, fever, flushing, headache, nausea and vomiting, trouble breathing, or weakness.

This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Tell your doctor right away if you have blurred vision, chills, confusion, cough, diarrhea, dizziness, faintness, or lightheadedness when getting up suddenly from a lying or sitting position, fast, pounding, or irregular heartbeat or pulse, fever, hives, itching, skin rash, nausea, runny nose, shivering, sneezing, sweating, tingling of the throat, trouble breathing, unusual tiredness or weakness, or watery eyes after you receive the medicine.

Check with your doctor right away if you have clay-colored stool, dark urine, decreased appetite, fever, itching, loss of appetite, nausea and vomiting, skin rash, stomach pain, swelling of the feet or lower legs, unusual tiredness or weakness, or yellow eyes or skin. These may be symptoms of a serious liver problem.

This medicine may increase risk for blood clots. Check with your doctor right away if you have pains in the chest, groin, or legs, especially the calves of the legs, severe headaches of sudden onset, sudden loss of coordination, sudden onset of trouble breathing for no apparent reason, sudden onset of slurred speech, or sudden vision changes.

While you are being treated with valoctocogene roxaparvovec-rvox injection, do not have any immunizations (vaccines) without your doctor's approval. Live virus vaccines should not be given with valoctocogene roxaparvovec-rvox.

Do not donate semen for at least 6 months after treatment.

Do not donate blood, organs, tissues or cells for transplant after treatment.

Do not take other medicines unless they have been discussed with your doctor. This includes prescription or nonprescription (over-the-counter [OTC]) medicines and herbal or vitamin supplements.

Side Effects of This Medicine:

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur:

More common

Back pain

blurred vision

chest tightness

chills

confusion

cough

diarrhea

difficulty swallowing

dizziness, faintness, or lightheadedness when getting up suddenly from a lying or sitting position

fast heartbeat

fever

flushing

headache

hives, itching, skin rash

nausea and vomiting

puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue

runny nose

shivering

sweating

tingling of the throat

trouble breathing

unusual tiredness or weakness

watery eyes

Less common

Loss of appetite

stomach pain

Incidence not known

Dark urine

pains in the chest, groin, or legs, especially the calves of the legs

severe headaches of sudden onset

sudden loss of coordination

sudden onset of slurred speech

sudden vision changes

yellow eyes or skin

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

General feeling of discomfort or illness

headache

unusual drowsiness, dullness, tiredness, weakness or feeling of sluggishness

Less common

Stomach pain

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.