Novartis experimental medicine shows improvement in spinal muscular atrophy

Investing.com — Novartis AG (SIX:) has reported that an advanced study showed their new experimental drug improved motor function in children suffering from a form of spinal muscular atrophy. The children involved in the study have a variant of the disease known as type 2, were at least two years old, could sit unaided but had never walked independently.

Novartis shares were little changed in early Zurich trading. Over the past year, the stock has seen a gain of 4.4%.

The new treatment shares the active ingredient with Novartis’s gene therapy Zolgensma, which was introduced five years ago as the first potential cure for babies with spinal muscular atrophy. The new medicine, however, would be administered differently and target older children.

Zolgensma, when it was launched, was priced at $2.1 million in the US, making it the first medicine to cost more than $1 million. The high cost of these therapies is justified by drugmakers due to their potential to save lives in rare diseases.

The Swiss pharmaceutical company 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.

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