Zolgensma’s approval was a notable milestone, making it both the second gene therapy approved in the U.S., and the second treatment for SMA, a potentially deadly disease for which no medicines existed until four years ago.
But it’s been a bumpy ride for Novartis since that 2019 approval. The FDA charged the company of manipulating preclinical data supporting its original application, a controversy that led Novartis to fire some top scientists and shake up its quality control operations. (The agency opted against sanctioning Novartis, however.)
