Valoctocogene Roxaparvovec Dosage

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.

Further information

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

Medical Disclaimer