The purpose of this site is to provide a global source of safety information from clinical trials and real-world data for healthcare professionals. This site will provide periodic updates on specific safety data that is of clinical interest. This site is NOT inclusive of all safety information including adverse events and/or risks associated with valoctocogene roxaparvovec treatment. It is NOT a source for real-time reporting of adverse events or real-time information of safety events.
Safety information provided on this site is from published manuscripts, congress oral presentations, posters, and abstracts. Data may vary based on the source, please refer to your local country product label for the full safety profile.
BioMarin continues to monitor long term safety via ongoing clinical studies, post approval studies, and ongoing registries. BioMarin will continue to provide safety and efficacy updates at relevant congresses through oral presentations, posters, and abstracts.
This website is updated at least every 6 months. The last update was 06/12/2023.
To date, valoctocogene roxaparvovec has been administered to more than 160 clinical participants with some having received an infusion 7 years ago. Please see the clinical trial program on the BioMarinMedical.com Hemophilia A Page for more information.