Novartis reports cases of tumours in children following treatment with Zolgensma
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Swiss pharmaceuticals giant Novartis has reported nine cases of tumours in children worldwide who had previously been treated with the gene therapy Zolgensma for a rare inherited muscle disorder. In two cases, there are indications of a link to the treatment.
The treatment, hailed as a medical breakthrough in 2019, is now coming under scrutiny due to potential safety risks.
Novartis said in a press release that nine reports of tumours in children treated with Zolgensma have been received since the therapy was first authorised in 2019.
In two cases, genetic material from the delivery vehicle used in the therapy – known as a vector – was detected in the patients’ DNA. Both children affected have been treated and remain under medical supervision.
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Whatever happened to the world’s most expensive drug?
Zolgensma is used to treat spinal muscular atrophy (SMA). In this rare genetic disorder, nerve cells die off, causing the muscles to become progressively weaker. What makes this therapy unique is that it is administered to children under the age of two as a single infusion, delivering a functional copy of the defective gene into the cells. It thus targets the very cause of the disease.
+ How can a drug cost $2.1 million?
Zolgensma also made headlines when it was launched in 2019 because of its price: at around $2.1 million (CHF1.7 million) per treatment, depending on the country and region, it was considered the world’s most expensive medicine at the time.
Further investigations under way
So far, the tumours of three of the nine affected children have been examined in more detail, as the British patient organisation SMA UK states on its website. While vector DNA integration was detected in two of these cases, there appears to be no link between the treatment and the tumour in the third child. Information regarding the affected children remains limited at this stage. Further details from the regulatory authorities are awaited.
Over 6,000 patients treated worldwide
According to Novartis, more than 6,000 patients worldwide have received Zolgensma since its authorisation as part of clinical trials, special access programmes and routine treatment. The company is continuously reviewing new safety information and all reported cases.
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Novartis emphasised that the potential risk of tumour formation due to the integration of vector DNA into the genome is already listed in the product information for Zolgensma.
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Translated from German, reviewed by an English Department journalist.
