The patients developed acute liver failure but the manufacturer of Zolgensma insists it’s not a new “safety signal”
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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.”
Meanwhile, Russian health regulator Roszdravnadzor announced on Friday that it had launched “a comprehensive assessment” of the circumstances which led to the death of a Zolgensma-treated patient.
The head of Russian SMA Families charity fund Olga Germanenko told RIA Novosti on Friday that not one but two patients had died in the country: one in 2021, before the medication was available in Russia, and another one in June this year.
“The first child received the drug through a lottery run by the manufacturer in countries where it was not available. The second child has already received therapy at the expense of the federal budget,” she said.
Germanenko also stressed that it is not possible to guarantee the safety of any medication and it is necessary to conduct an assessment to establish if there was a direct connection between use of Zolgensma and the deaths.
Spinal muscular atrophy is a genetic disease which leads to weakening and eventual failure of the muscles. Babies born with severe forms of the disease usually die before they turn two. Zolgensma was approved in the US in 2019 for use in under-twos and won conditional EU approval in early 2020. The Russian Health Ministry registered the treatment, which is used as one-time intravenous injection, in December last year. Zolgensma is one of just three existing medications for SMA.
