Swiss pharmaceutical company Novartis has reported nine cases of tumors in children who received the gene therapy Zolgensma. This is a treatment used against the rare disease spinal muscular atrophy (SMA). The cases were identified in patients who have received this therapy since 2019, when it was introduced.
The news has raised concern and is being examined by health authorities. They are evaluating the new data to determine whether there is a risk associated with the use of the treatment. It is also being examined whether additional measures are needed for patient safety.
Zolgensma is produced by Novartis and is considered one of the most expensive therapies in the world, with a price of about 1.7 million Swiss francs for a single dose. It is used in children diagnosed with spinal muscular atrophy, a genetic disease that affects the nerve cells responsible for controlling muscles.
This disease can cause severe muscle weakness. Since the therapy was introduced in 2019, more than 6,000 patients worldwide have received Zolgensma. According to the reporting, of the nine tumor cases detected, genetic evidence suggesting a possible link to the therapy was identified in two of them.
However, this does not mean it has been proven that the treatment caused the tumors. The matter is being examined by the relevant health authorities. They are analyzing the new data to determine whether there is a risk associated with the use of this treatment.
Likewise, it is being examined whether additional measures are needed for patient safety. Zolgensma is considered one of the important advances in treating rare genetic diseases. But its high price and now concerns about its safety have raised questions about its widespread use.
Regulatory authorities are evaluating the new data to determine whether there is a risk associated with the use of this treatment. It is also being examined whether additional measures are needed for patient safety. Until the review is concluded, it remains unclear whether the therapy will face new restrictions. It is also not known whether it will continue to be used without changes.