Two children have died from acute liver failure after taking world’s most expensive drug. Although the patients had acute liver failure, Zolgensma’s manufacturer claims that this is not a new “safety signal.”
After receiving Zolgensma gene therapy, sometimes referred to as the most costly drug in the world, two infants died of acute liver failure, the drug’s maker Novartis reported on Thursday.
According to the Swiss pharmaceutical company, acute liver failure is a “known adverse event” of the medication used to treat patients with spinal muscular atrophy (SMA), however these are the first death cases brought on by this complication. According to Novartis, the patients had taken corticosteroids to prevent serious liver damage.
According to the company, the deaths happened in Russia and Kazakhstan. It also noted that it had updated medical personnel as well as informed health authorities in the areas where the drug is supplied. 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.
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Even though the fatal cases are “important safety information,” Novartis highlighted that they are not regarded as “a new safety signal” because they “firmly believe in the overall favorable risk/benefit profile of Zolgensma.” According to the pharmaceutical business, more than 2,300 individuals have already received treatment with the medicine “across clinical trials, managed access programs, and in the commercial setting.”
In the meantime, the Russian health regulator Roszdravnadzor declared on Friday that it has begun “a comprehensive assessment” of the events that resulted in the death of a patient receiving Zolgensma.
The head of the charity fund for Russian SMA Families, Olga Germanenko, revealed to RIA Novosti on Friday that just not one but two patients had passed away in the nation: one in 2021, before the treatment was made available there, and another in June of 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 emphasized that no medication can be guaranteed to be safe, and that an investigation is required to determine whether there was a causal link between the use of Zolgensma and the fatalities.
Spinal muscular atrophy is a genetic condition that causes the muscles to deteriorate and eventually collapse. Most newborns with severe types of the illness pass away before they are two. In the US, Zolgensma received approval in 2019 for use in children under two, while in the EU, it received conditional approval in early 2020. The procedure, which involves a single intravenous injection, was authorized by the Russian Health Ministry in December of the previous year. There are only three drugs available for SMA, including zolgensma.