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FDA Approves Hemophilia A Gene Transfer Rx

Today, July 3, 2023, the FDA approved the use of BioMarin’s Roctavian┬« [valoctocogene roxaparvovec] for treating adults with severe hemophilia A without inhibitors. The FDA also approved BioMarin’s companion diagnostic, AAV5 DetectCDx, which identifies the patients most likely to benefit from Roctavian. Click here for the LinkedIn announcement.

Click here for a provocative FirstWord PHARMA discussion of the new therapeutic.

The European Medicines Agency gave conditional marketing approval for BioMarin’s therapeutic on June 24, 2022.

Comments (1)
Bleeding Disorders
Dr. Emmanuel Favaloro
Aug 9, 2023 7:50pm

As we move forward to the Brave New World, I recommended reading: Valentino LA, Kaczmarek R, Pierce GF, Noone D, O’Mahony B, Page D, Rotellini D, Skinner MW. Hemophilia gene therapy: first, do no harm. J Thromb Haemost. 2023 Jun 21:S1538-7836(23)00501-9. doi: 10.1016/j.jtha.2023.06.016. Epub ahead of print. PMID: 37353081

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