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The pharmaceutical company Novartis is headquartered in Switzerland
Photo: Arnd Wiegmann / REUTERS
Two children have died of acute liver failure after being treated with Novartis' gene therapy drug Zolgensma.
The pharmaceutical company confirmed this in a statement on Friday.
"After two recent deaths and in accordance with the health authorities, we will update the label to indicate that fatal acute liver failure has been reported," the group said, according to dpa.
The drug costs around $2 million
The release continues, “We strongly believe in the overall favorable benefit-risk profile of Zolgensma, which to date has treated more than 2,300 patients worldwide in clinical trials, managed access programs and commercial settings became".
Acute liver failure is a known side effect that has been reported after treatment with Zolgensma.
It is already highlighted in the product labeling.
The deaths occurred in Russia and Kazakhstan.
Both patients had been treated with steroids, which are used to minimize risk, a few weeks after Zolgensma therapy.
According to Novartis, these are the first fatal cases of acute liver failure.
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Zolgensma is used to treat spinal muscular atrophy, a rare inherited disorder that causes muscle wasting and is often fatal by the age of two in its most severe form.
According to the Novartis subsidiary Avexis, the drug aims to combat the genetic cause of spinal muscular atrophy by replacing the function of the missing or non-functioning SMN1 gene with the therapy.
The therapy is administered intravenously once and brings a new working copy of the SMN1 gene into the patient's cells, which should stop the progression of the disease.
The drug was approved in the United States in May 2019.
It can also be used in the EU.
Priced at around $2 million, Zolgensma is also the most expensive single-use therapy in the world.
In addition to Zolgensma, there are only two other drugs from Biogen and Roche that have been approved for the treatment of spinal muscular atrophy.
According to Avexis, around 500 to 600 children with spinal muscular atrophy are born in Europe every year.
With regard to the Zolgensma gene therapy, discussions about possible safety risks have repeatedly arisen.
mar/dpa
