Roctavian (valoctocogene roxaparvovec-rvox) — Medica

Initial criteria

• Patient is male
• age ≥ 18 years
• Patient has not received Roctavian in the past
• Patient has severe hemophilia A as evidenced by a baseline uncorrected Factor VIII level < 1 IU/dL
• Patient does not have detectable pre-existing antibodies to adeno-associated virus 5 (AAV5) by an FDA-approved test
• Patient has a history of use of Factor VIII therapy for at least 150 exposure days
• Factor VIII inhibitor titer testing has been performed within the past 30 days AND patient does not currently have an inhibitor to Factor VIII AND patient does not have a history of Factor VIII inhibitors
• Prophylactic therapy with Factor VIII will not be given after Roctavian administration once adequate Factor VIII levels have been achieved (episodic therapy is acceptable for bleeds or procedures)
• Patient does not have a known hypersensitivity to mannitol
• Patient does not have chronic or active hepatitis B
• Patient does not have active hepatitis C
• Patient is not HIV positive
• Patient does not have evidence of significant hepatic fibrosis or cirrhosis
• Patient has had liver function testing in the past 30 days showing alanine aminotransferase, aspartate aminotransferase, total bilirubin, alkaline phosphatase, gamma-glutamyl transferase ≤ 1.25× upper limit of normal and INR < 1.4 OR a hepatologist has determined Roctavian use is clinically appropriate if lab values exceeded these limits
• Platelet count within past 30 days ≥ 100 × 10⁹/L
• Creatinine level within past 30 days < 1.4 mg/dL
• Medication is prescribed by a hemophilia specialist physician
• Current patient body weight has been obtained within past 30 days
• If above criteria are met, approve one dose of Roctavian 6×10¹³ vector genomes/kg by intravenous infusion as a one-time (per lifetime) single dose

Approval duration

one-time (per lifetime)