(Bloomberg) -- Novartis AG said a new experimental medicine improved motor function in children with a form of spinal muscular atrophy, the devastating disorder targeted by its gene therapy Zolgensma, in an advanced study.
The study included children with a form of the disease known as type 2 who had never received treatment, were aged two or older and could sit but had never walked independently, the Swiss drugmaker said.
The new treatment, which shares Zolgensma’s active ingredient, could extend Novartis’s offering in a lucrative niche of life-saving therapies for rare diseases. Zolgensma, introduced five years ago, was the first potential cure for babies with spinal muscular atrophy. The new medicine would be administered differently and target older children.
When introduced, Zolgensma was priced at $2.1 million in the US, making it the first medicine to cost more than $1 million. Drugmakers can charge far more for such therapies than for routine treatments.
Novartis said it plans to present detailed results of the trial at a medical meeting next year and discuss the findings with regulators.
The drug was compared to a sham control, a procedure designed to mimic the administration of an experimental medicine that doesn’t deliver the active treatment.
Spinal muscular atrophy occurs when babies are born with a flawed or absent gene needed to make a protein key to nerve cells’ survival. It affects muscle function, including breathing, swallowing and basic movement.
Novartis shares were little changed in early Zurich trading. The stock has gained 4.4% in the past year, outperforming Swiss rival Roche Holding AG and the Bloomberg Europe Pharmaceutical Index.
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