Valoctocogene Roxaparvovec Infusion Suppliers & Bulk Manufacturers

Available Forms: Intravenous infusion

Available Strengths: A single-dose gene therapy designed to increase factor VIII production in hemophilia A patients

Reference Brands: Roctavian(US & EU)

Category: Cell & Gene Therapies

Valoctocogene roxaparvovec, a one-time AAV5 gene therapy infusion for adults with severe haemophilia A without inhibitors or AAV5 antibodies. For qualified haemophilia treatment centres via the authorisation holder.

Product Description:

Valoctocogene roxaparvovec is a one-time gene therapy for severe haemophilia A. It uses a non-replicating adeno-associated virus serotype 5 (AAV5) vector carrying a B-domain-deleted human factor VIII gene under a liver-specific promoter. After a single intravenous infusion, liver cells start making factor VIII themselves. The dose is set in vector genomes per kilogram of body weight rather than as a fixed strength, and the frozen suspension is thawed and prepared in the pharmacy for each patient.

It is intended for adults with severe haemophilia A who have no history of factor VIII inhibitors and no detectable antibodies to AAV5, which a validated test confirms before treatment. Raised liver enzymes are common in the first months and are managed with corticosteroids or other immunosuppressants, so patients need frequent liver tests and factor VIII activity checks for a long period. Factor VIII levels vary between patients and can fall over time, so some return to prophylaxis. Labels also set alcohol limits and contraception rules after dosing.

Sourcing notes. This gene therapy is not traded through ordinary wholesale channels. It reaches patients only through the authorisation holder's controlled distribution to qualified haemophilia treatment centres, or through named-patient routes where the law allows. Vials are shipped and stored frozen at ultra-low temperature, so the centre needs a monitored freezer and staff trained in thawing. Genetically modified organism rules may apply to handling and waste. A centre should first confirm access to the AAV5 antibody test, the liver monitoring plan and long-term follow-up, and ask the authorisation holder about status in its country.