Zolgensma

Two children die after taking world’s ‘most expensive drug’
The patients developed acute liver failure but the manufacturer of Zolgensma insists it’s not a new “safety signal”
Two children died of acute liver failure after being treated with Zolgensma gene therapy, which is often referred to as the most expensive drug in the world, the medication’s manufacturer Novartis said on Thursday.

Acute liver failure is a “known adverse event” of the drug, which is used in patients suffering from spinal muscular atrophy (SMA), the Swiss pharmaceutical company said, but these are the first fatal cases caused by this complication. The patients had received corticosteroids, Novartis said, to prevent severe liver damage.

The deaths occurred in Russia and in Kazakhstan, the company said, adding that it had notified health authorities in markets where the drug is sold and has updated healthcare workers as well. Zolgensma costs about $2 million per patient.

“Pending health authority review and approval, the Zolgensma label will be updated to inform prescribers that fatal acute liver failure has been reported,” the manufacturer said.

Novartis emphasized that while the lethal cases are “important safety information,” they are not considered “a new safety signal” as the company continues to “firmly believe in the overall favorable risk/benefit profile of Zolgensma.” The pharmaceutical company said the drug has already been used to treat more than 2,300 patients worldwide “across clinical trials, managed access programs, and in the commercial setting.”