Valoctocogene Roxaparvovec Dosage

Medically reviewed by Drugs.com. Last updated on Apr 8, 2024.

Usual Adult Dose for Hemophilia A

6 × 10(13) vg/kg as a single IV infusion at a rate of 1 mL/min

Duration of therapy: Once

Comments:

• 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

Renal Dose Adjustments

No adjustment recommended.

Liver Dose Adjustments

No adjustment recommended.

Precautions

CONTRAINDICATIONS:

• Active infections
• Hepatic fibrosis or cirrhosis
• Hypersensitivity to mannitol

Safety and efficacy have not been established in patients younger than 18 years.

Consult WARNINGS section for additional precautions.

Dialysis

Data not available

Other Comments

Administration advice:

• Prior to dosing this drug, patients should be assessed for:
• absence of factor VIII inhibitors
• absence of antibodies to AAV5
• liver health status
• hepatic fibrosis
• suitability for corticosteroids and/or other immunosuppressive therapy may be evaluated
• If these tests are abnormal, the patient may not be a suitable candidate for this gene therapy.
• If liver abnormalities and/or liver function test abnormalities are detected, consult a hepatologist to assess eligibility for this drug.
• Administer this drug under the supervision of a physician experienced in the treatment of hemophilia and/or bleeding disorders.
• Do not use filter needles to extract this drug from vials.
• Do not administer as an intravenous push or bolus.
• Do not use a central line or port.
• Infuse the suspension through a suitable peripheral vein using an infusion catheter with an in-line filter and a programmable syringe pump.
• The infusion should be completed within 10 hours of the initial drug product thawing.
• Do not infuse this drug concomitantly in the same IV line with other agents.
• Maintain venous access during the subsequent observation period.
• The manufacturer product information should be consulted for more information on calculating the dose and number of vials needed.

Storage requirements:

• Store upright at -60C (minus 76F) or less in a carton until ready to use.
• Protect from light.
• During preparation and administration, after thawing, this drug can be kept at room temperature for a maximum of 10 hours, including preparation and infusion times.
• Store thawed intact vial upright at 2 to 8C and protect from light for up to 3 days (in the original carton).
• Do not refreeze.
• Treat spills with virucidal agent with proven activity against non-enveloped viruses and blot using absorbent materials.

Preparation technique:

• The manufacturer product information should be consulted.

IV compatibility:

• The manufacturer product information should be consulted.

General:

• Perform baseline testing to select patients for treatment with this drug.
• For information on US FDA-approved tests visit: http://www.fda.gov/CompanionDiagnostics.
• Advise patients to contact BioMarin Pharmaceutical Inc. (1-866-906-6100) if they are diagnosed with any malignancy.
• There is limited information on the safety and effectiveness of this drug in patients with HIV infection.

Monitoring:
Hepatic: Liver enzymes, ALT
Hematologic: Factor VIII activity, factor VIII inhibitors
Oncologic: Malignancy, hepatocellular carcinoma

Patient advice:

• Read the US FDA-approved patient labeling (Patient Information).
• Infusion reactions including anaphylaxis may occur.
• Immediately notify medical staff if symptoms indicative of infusion reactions or thrombotic events develop.
• Seek medical attention in case of a new or recurrent reaction.
• This drug may cause an elevation of certain liver enzymes. Weekly blood test may be needed for at least 26 weeks to monitor liver enzyme levels.
• Tapering factor VIII concentrates and hemostatic agents is encouraged.
• Do not consume alcohol for at least one year after treatment.
• Confirm with your health care provider prior to using any medications, herbal products, or supplements.
• Elevated factor VIII activity above the upper limit of normal may occur, which may increase the risk of a thromboembolic event.
• For 6 months after administration, men must not donate semen, and male patient and/or female partner must avoid or postpone pregnancy.
• Use adequate methods of contraception.
• Do not donate blood, organs, tissues, and cells for transplantation after treatment with this drug.

See also:

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Medical Disclaimer