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.
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