Swiss pharmaceutical giant Novartis has warned health authorities of the deaths of two children treated with Zolgensma, a gene therapy for a rare and often fatal condition for which there were no treatment options until the drug arrived. , he said on Friday.
These two deaths occurred in Russia and Kazakhstan, said the Swiss group in an email to AFP.
These two children died of acute liver failure five to six weeks after receiving this treatment and one to ten days after the administration of corticosteroids which must be dispensed afterwards to reduce the adverse effects, he explains.
Novartis has notified the two deaths to health authorities in all countries where the treatment is marketed, including the US health agency, the Food and Drug Administration (FDA), which was the first to approve it.
Zolgensma is a gene therapy for spinal muscular atrophy, a rare but serious and incurable genetic disease until this treatment was developed.
Babies affected by this disease quickly lose motor neurons for essential muscle functions, such as breathing, swallowing, speaking and walking. Without treatment, their muscles weaken and the disease gradually progresses to paralysis or death, often before the second birthday.
This gene therapy has revolutionized the treatment of this degenerative disease which condemned babies to an early death. It makes it possible to directly attack the genetic root of the disease.
It was first approved in the United States in 2019, but its launch was accompanied by a lot of controversy, including its cost. In the United States, Zolgensma cost $2 million when it was launched, making it the most expensive treatment in the world.
This cost is explained by the manufacturing complexity of the treatment, which consists of introducing genetic material into cells to correct defective genes.
Acute liver failure is one of the known potential side effects, explains the Swiss laboratory, stressing that this risk is already indicated in the warnings detailing the information on the treatment. However, the group intends to update the information notice following these two deaths.
Novartis nevertheless said it continued to “firmly believe” in this treatment with regard to the risks for patients affected by this disease. Globally, Zolgensma has been used to treat over 2,300 patients since its launch.