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This website was developed and funded by BioMarin and is intended for healthcare professionals only. It offers a non-exhaustive selection of materials, including but not limited to publications, presentations, posters and educational materials, to support scientific exchange.
Clinical study results reflect knowledge at the study’s completion and may not be current. Review the methodology, limitations, and authors’ financial disclosures for details.
The information on this website is not medical advice and should not replace independent medical judgment or further research. Content on investigational therapeutics or uses of approved products does not confirm safety or efficacy. BioMarin does not endorse off-label use of its medicines.
As product information may vary by country, please refer to your local health authority regulations and respective approved product information for indications and safety details.
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.
The website was last updated March 2024 and may not reflect all available information. Please reach out to Medical Information for additional information.
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.
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.
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 medinfo@bmrn.com or complete a request form to inquire about this specific material.
Prescribing Information
This site focuses on specific safety data that is of clinical interest and is not inclusive of all adverse events and is not a source for real-time reporting of adverse events and safety information. Please refer to the product label for full safety profile.
You may report adverse events to BioMarin at drugsafety@bmrn.com. You are encouraged to report negative adverse events of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.