FDA heaps further misery on Novartis’s Zolgensma

The FDA has thrown another roadblock into the path of Novartis's intrathecal version of Zolgensma, by requesting a further pivotal study. This follows a separate partial clinical hold, under which this formulation of the gene therapy was placed in late 2019, and is the latest embarrassing misstep in the development of the product, following a data manipulation scandal and delays to approvals. The intrathecal version of Zolgensma is intended to extend the spinal muscular atrophy treatment into an older patient population – an intravenous formulation is approved in babies up to two years old – and allow the Swiss group to start competing with Biogen, the maker of Spinraza. Wider approval will be needed if Zolgensma is to achieve its consensus forecast sales of $2.3bn in 2024, and this looks ambitious considering that the new pivotal study cannot be started until the clinical hold is lifted. Analysts believe the new formulation might not get to market until the end of 2023. This postponement will prove costly to Novartis, as it could allow Roche’s recently approved Evrysdi to gain substantial market share. Not only is Evrysdi cheaper than its rivals, it is suitable for all ages and severities of disease, and taken orally.