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FDA slams brakes on trial for world's most expensive drug following safety concerns in children

Published on 30/10/19 at 12:00pm

The FDA has temporarily shut down a clinical trial evaluating Zolgensma – the world’s most expensive drug at $3.1 million – after safety concerns were raised during study in animals.

Novartis, the manufacturer of the therapy, raised the alarm to the authorities after the study revealed instances of dorsal root ganglia (DRG) mononuclear cell inflammation in animal subjects – a condition neurological in nature that can lead to nerve damage or loss.

The findings could have significant implications on further human tests – Novartis was aiming to investigate the impact of a higher dose of the drug in paediatric patients up to the age of five years old with spinal muscular atrophy.

However, it is not known whether the future of the drug will be affected by the discovery. “We need to better understand, what the issues are and whether there really is a read-through to the human trial,” remarked analyst Stefan Schneider.

Novartis responded to the news: “The clinical significance of the DRG inflammation observed in this pre-clinical animal study is not known and was not seen in prior animal studies. We are working with health authorities to confirm further guidance to clinical investigators.”

Matt Fellows

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