Novartis Wins Big: Gene Therapy Drug Gets FDA Green Light

Novartis just scored a regulatory W—the FDA approved Itvisma for spinal muscular atrophy (SMA), sending the stock up 3% while the broader market only climbed 0.9%.

What’s the play?

Itvisma is a gene replacement therapy for SMA patients aged 2+. It’s basically a new formulation of their existing pediatric SMA drug Zolgensma, backed by phase 3 trial data showing solid improvements in motor function and disease stabilization.

The company’s positioning this as the first and only gene replacement option for the condition—a meaningful differentiation in a market where SMA affects an estimated 10,000-25,000 patients in the US alone.

Why this matters

Gene therapies have historically been a graveyard for investors. Watching one actually make it through the FDA approval gauntlet and move the needle on stock price is rare enough to grab attention. For Novartis specifically, Itvisma expands their addressable market beyond pediatric cases, which could open up revenue streams in an underserved adult population.

The risk? Gene therapy pricing and real-world uptake remain unpredictable. But on the regulatory front, this is a clean win.