ROCTAVIAN Solution for infusion Ref.[50172] Active ingredients: Valoctocogene roxaparvovec

Source: European Medicines Agency (EU)  Revision Year: 2023  Publisher: BioMarin International Ltd., Shanbally, Ringaskiddy, County Cork, P43 R298, Ireland

Product name and form

ROCTAVIAN 2 × 1013 vector genomes/mL solution for infusion.

Pharmaceutical Form

Solution for infusion.

A clear, colourless to pale yellow solution with a pH of 6.9-7.8 and an osmolarity of 364-445 mOsm/L.

Qualitative and quantitative composition

Valoctocogene roxaparvovec is a gene therapy medicinal product that expresses the B-domain deleted SQ form of human coagulation factor VIII (hFVIII-SQ). It is a non-replicating recombinant adeno-associated virus serotype AAV5 based vector containing the cDNA of the B-domain deleted SQ form of human coagulation factor VIII gene under the control of a liver-specific promoter.

Valoctocogene roxaparvovec is produced in a baculovirus expression system that derived from cells of Spodoptera frugiperda (Sf9 cell line) by recombinant DNA technology.

Each mL of valoctocogene roxaparvovec solution for infusion contains 2 × 1013 vector genomes.

Each vial contains 16 × 1013 vector genomes of valoctocogene roxaparvovec in 8 mL solution.

Excipient with known effect: This medicinal product contains 29 mg sodium per vial.

For the full list of excipients, see section 6.1.

Active Ingredient Description
Valoctocogene roxaparvovec

Valoctocogene roxaparvovec is an adeno-associated virus serotype 5 (AAV5) based gene therapy vector causing the expression of the B-domain deleted SQ form of a recombinant human factor VIII (hFVIII-SQ) under the control of a liver-specific promoter. The expressed hFVIII-SQ replaces the missing coagulation factor VIII needed for effective haemostasis.

List of Excipients

Disodium phosphate dodecahydrate (E339)
Mannitol (E421)
Poloxamer 188
Sodium chloride
Sodium dihydrogen phosphate dihydrate (E339)
Water for injections

Pack sizes and marketing

10 mL vial (cyclic olefin polymer plastic resin) with a stopper (chlorobutyl rubber with fluoropolymer coating), crimp seal (aluminium) and flip-off cap (polypropylene) containing 8 mL of solution for infusion.

Each carton contains 1 vial.

Marketing authorization holder

BioMarin International Ltd., Shanbally, Ringaskiddy, County Cork, P43 R298, Ireland

Marketing authorization dates and numbers

Date of first authorisation: 24 August 2022
Date of latest renewal: 24 July 2023

Drugs

Drug Countries
ROCTAVIAN Austria, Estonia, France, Ireland, Italy, Lithuania, Poland, United States

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