Roctavian

Generic name: Valoctocogene Roxaparvovec-rvox
Dosage form: injection for infusion
Drug class: Miscellaneous coagulation modifiers

Usage of Roctavian

Roctavian (valoctocogene roxaparvovec-rvox) is a single-dose gene therapy infusion that may be used to treat adults with severe hemophilia A who have had a blood test to determine they do not have antibodies to a particular adenovirus serotype, AAV5.

Roctavian uses a modified virus, called a vector, to deliver a working copy of the factor VIII gene to liver cells to enable the body to produce clotting factor on its own, which helps the blood to clot and prevents or reduces the occurrence of bleeding. The modified virus does not contain viral DNA and does not cause disease in humans.

Roctavian was FDA approved on June 29, 2023.

Roctavian side effects

Your doctor will provide emergency medical help if you have signs of an allergic reaction to Roctavian such as hives; difficulty breathing; swelling of your face, lips, tongue, or throat.

Serious side effects of Roctavian include:

Hepatotoxicity

Serious infusion-related reactions

Thromboembolic events.

The most common side effects of ROCTAVIAN are:

Abdominal pain

Fatigue

Headache

Infusion-related reactions

Nausea

Vomiting.

Changes to laboratory results have also been reported – some of these are consistent with how Roctavian works in your body.

These are not all the possible side effects of ROCTAVIAN. Talk to your doctor for medical advice about side effects. You may report side effects to BioMarin Pharmaceutical Inc. at 1-866-906-6100 or FDA at 1-800-FDA-1088.

Before taking Roctavian

You should not receive Roctavian if you are allergic to mannitol or any of the other ingredients in the infusion.

Make sure all your vaccinations are up to date before receiving Roctavian.

To make sure Roctavian is safe for you, tell your doctor if you have:

any general risk factors for unwanted blood clots and cardiovascular disease, such as oral contraceptive use, history of deep vein thrombosis (DVT), high blood pressure or cholesterol, are overweight

HIV infection

lowered immunity

inhibitors or a history of inhibitors to factor VIII

a female partner that plans to become pregnant within 6 months of treatment. The vector DNA is detectable for at least 182 days post-administration.

Roctavian is not intended for administration in women.

There are no data on the use of Roctavian in pregnant or breastfeeding women.

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How to use Roctavian

Roctavian is administered as a single, one-time infusion that is given into a vein in a medical facility by a healthcare professional.

The recommended dosage is 6 x 1013 vector genomes per kilogram of body weight. For example, a 70kg person should receive 210mL of Roctavian which equates to 27 vials (each vial contains 16 × 1013 vg in 8mL). Round up to the nearest vial.

The infusion will take 2 to 5 hours or longer depending on your weight and how you respond to the infusion.

During and after the infusion, you will be monitored for any possible side effects. Your healthcare provider will determine when you can go home (usually later the same day).

It is not yet known if Roctavian or other gene therapy treatments can be repeated.

After treatment with Roctavian, you will be asked to enroll in a 15-year registry to help study the long-term safety of the treatment and how well it continues to work.

While the majority of patients experience a benefit from Roctavian, the response to treatment can vary and some people do not experience a benefit. Your doctor will monitor your lab tests and talk to you about whether you can stop prophylaxis, whether you should start prophylaxis again, and whether and how you should treat any surgeries, procedures, injuries, or bleeds.

Warnings

Do not take Roctavian if you:

have an active infection or if you have a long-term infection that is not controlled by the medicines you take

have scarring of the liver (significant liver fibrosis or cirrhosis)

are allergic to mannitol (an inactive ingredient in Roctavian).

May cause infusion-related reactions, including hypersensitivity reactions and anaphylaxis. You will need to be monitored for at least 3 hours after Roctavian administration. If you develop symptoms your doctor will slow or interrupt administration and give appropriate treatment, and restart at a slower rate once symptoms resolve. The infusion will be discontinued if anaphylaxis occurs.

Your doctor will monitor your liver function, specifically alanine aminotransferase (ALT) weekly for at least 26 weeks and institute corticosteroid treatment in response to ALT elevations as required. Continue to monitor ALT until it returns to baseline. Factor VIII activity levels and other laboratory values will also be monitored since ALT elevation may be accompanied by a decrease in factor VIII activity.

Thromboembolic events may occur with elevated factor VIII activity above the upper limit of normal (ULN), which has been reported following Roctavian infusion. People with risk factors for thrombosis including cardiovascular risk factors are more at risk.

Annual liver ultrasound is important in people with risk factors for liver cancer such as hepatitis B or C, non-alcoholic fatty liver disease, chronic alcohol consumption, non-alcoholic steatohepatitis, and advanced age). Alpha-fetoprotein testing will be performed following administration.

What other drugs will affect Roctavian

No interaction studies have been performed with Roctavian. Before administration, review all existing medications to see if they should be modified to prevent anticipated interactions, including their effect on liver function and clotting risk. The following medications are not recommended with Roctavian:

efavirenz

isotretinoin

live vaccines.

Agents that reduce or increase the plasma concentration of corticosteroids (such as those that induce cytochrome P450 3A4 can decrease the efficacy of the corticosteroid regimen used with Roctavian or increase their side effects.

When a new medication is started, close monitoring of liver function tests and factor VIII activity is recommended (such as weekly to every 2 weeks for the first month) to assess potential effects on both levels.

Not all possible interactions are listed here. Tell your doctor about all your current medicines and any medicine you start or stop using or intend to use, including prescription and nonprescription drugs, vitamins, herbal supplements, and vaccines.

Disclaimer

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