US News: Novartis issues security information to the most expensive medication in the world: Two children die in treatment with the most expensive drug in the world

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Two children died after treatment with Zohnensma, the most expensive drug produced by Novartis. Novartis names organ failure as the cause of death. The pharmaceutical company remains convinced of the “overall favorable risk-benefit profile of Zolgensma”.

Zwei Kinder sterben bei Behandlung mit teuerstem Medikament der Welt © provided by Blick Two children die during treatment with the most expensive drug in the world

The gene therapy Zöngensma produced by the Swiss pharmaceutical company is considered the most expensive drug in the world. A single injection into the spinal cord costs the equivalent of around two million francs. Novartis reported on Thursday that two patients died of acute liver failure after treatment with Zolgensma.

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Novartis notified the health authorities of the markets in which the drug is approved. "Although this is an important security information, but not a new security signal," said the Basel pharmaceutical company in a statement that Reuters has. Novartis "believes in the overall favorable risk benefit profile of Zengensma". Nevertheless, "the labeling will be updated to point out that liver failure has been reported on fatal acute acute."

Liver failure several weeks after the infusion

The victims are two children in Russia and in Kazakhstan. The fatal organ failure was "after a five- to six-week umensma infusion", a few days after the start of corticosteroid treatment. According to Novartis, the first fatal cases of acute liver failure are.

Zengensma has been approved in the Russian Federation since the end of 2021. The drug for children aged less than two years is currently one of three medication worldwide that are used to treat spinal muscle atrophy. This is a rare hereditary disease that often ends fatally at the age of two.

Zengensma was approved in the United States in May 2019 and is only the second genetic therapy for an inherited disease approved by the FDA. In addition to Zollgensma, there are only two other medication from Biogen and Roche, which have so far been approved for the treatment of spinal muscle atrophy. (Kes/Lui)

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