Valoctocogene Roxaparvovec: First Approval
Related Experiment Video
Updated: Aug 26, 2025

Author Spotlight: Addressing Regulatory Gaps in Molecular Studies by Quantifying Viral Vectors in Complex Matrices
Published on: July 14, 2023
1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.
Abstract:
Valoctocogene roxaparvovec (ROCTAVIAN™) is a gene therapy being developed by BioMarin Pharmaceutical Inc. for the treatment of haemophilia A. In August 2022, valoctocogene roxaparvovec was granted conditional marketing authorization in the EU for the treatment of severe haemophilia A [congenital factor VIII (FVIII) deficiency] in adults without a history of FVIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5). This article summarizes the milestones in the development of valoctocogene roxaparvovec leading to this first approval for severe haemophilia A.
More Related Videos
06:14Optimized LC-MS/MS Method for the High-throughput Analysis of Clinical Samples of Ivacaftor, Its Major Metabolites, and Lumacaftor in Biological Fluids of Cystic Fibrosis Patients
Published on: October 15, 2017
06:16Subconjunctival Administration of Adeno-associated Virus Vectors in Small Animal Models
Published on: March 16, 2022
Related Concept Videos
Prescription, Nonprescription and Orphan Drugs
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
Pharmacovigilance
This process, termed pharmacovigilance, aims to detect, evaluate, and minimize harmful effects related to medication use. The data collection for pharmacovigilance depends on spontaneous reporting systems, where healthcare professionals or patients voluntarily report suspected ADRs.
In some cases, there...
Drug Regulation
Preclinical Development: Overview
Gene Therapy
Targeted Cancer Therapies
There are several types of targeted therapies against...