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BioMarin’s enduring commitment to communicate safety information on valoctocogene roxaparvovec

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 Hemophilia A Page for more information.

Select Safety information provided below represents key safety areas of clinical interest with regards to valoctocogene roxaparvovec treatment. 

Click on each area for additional information.

Liver Health

Malignancy (vector integration)


FVIII Inhibitor Development

Recent Congress Posters and Presentations that contain updates on safety information

Access posters and presentations from recent congresses. All displayed posters and presentations are protected by copyright and are intended for individual use only. Please Note: Poster content may be unavailable or delayed. This could be due to copyright or embargo guidelines. You may check back later or contact or complete a request form to inquire about this specific material.

You may report adverse events to BioMarin at You are encouraged to report negative adverse events of prescription drugs to the FDA. Visit, or call 1-800-FDA-1088.