Novartis is in for a big month

Over the next few weeks, we’ll find out whether Novartis’s gene therapy for spinal muscular atrophy can win FDA approval for severe patients —and — whether it can benefit those with milder forms of the muscle-wasting disease.

That makes May a major test for Vas Narasimhan, the Novartis CEO who spent $8.7 billion of his company’s cash to get that gene therapy. But, as STAT’s Matthew Herper reports, winning approval is just the first step.

Novartis’s treatment, called Zolgensma, is expected to cost as much as $5 million for a one-time dose, which would make it the most expensive therapy in the world. If it’s going to be widely used, insurance plans will need alternative ways to pay that don’t just involve putting down a huge sum of money upfront, something of which Narasimhan is well aware.

“I believe it’s very important that we get these models set up, because we will enter a world where not just Novartis, but many companies will come with these therapies,” he said.

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