The first gene therapy to launch in the U.S., Roche’s Luxturna, gained FDA approval less than three years ago. The agency’s experience with these types of treatment, therefore, is more limited than with other drug classes.
The relative ease with which Luxturna, and then Novartis’ Zolgensma, passed regulatory muster may have resulted in misplaced confidence among investors that development of other gene therapies would be as smooth.
Revelations that Novartis’ application for approval of Zolgensma contained altered animal testing data, and the FDA’s subsequent investigation, have now been followed by closer reviews of several companies’ data packages.
