Zolgensma, a one-off treatment for infants born with a rare and often fatal genetic disease, came to the market five years ago at an eye-watering $2.1 million. But its Swiss manufacturer Novartis is now under growing pressure as annual sales decline and new markets prove tough to crack. When Novartis CEO Vas Narasimhan presented the company’s 2023 annual results in January, Zolgensma didn’t even get a mention. Launched with great fanfare in 2019, it was the first gene therapy to treat spinal muscular atrophy (SMA) – a rare neuromuscular disorder and the leading genetic cause of infant mortality worldwide. The one-time infusion was originally touted as a potential cure for SMA and one of the Swiss pharmaceutical giant’s six major growth drivers with multibillion-dollar sales potential. The consensus estimate among analysts at the time was that sales could reach $1.9 billion (CHF1.7 billion) a year, although one investment bank’s forecast was as high as $2.8 billion. In 2021, sales ...
