Novartis' Experimental Drug Shows Improvement in Spinal Muscular Atrophy

Investing.com -- Novartis AG reported that its new experimental drugs improved motor functions in children suffering from a form of spinal muscular atrophy in an advanced study. The children included in the study had type 2 variant of the disease, were at least two years old, could sit unassisted but had never walked independently.

Novartis shares remained largely unchanged in early trading in Zurich. Over the past year, the stock has seen a 4.4% increase.

The new treatment shares the same active ingredient as Zolgensma, the gene therapy launched by Novartis five years ago as the first potential treatment for infants with spinal muscular atrophy. However, the new drug will be administered differently and aims to target older children.

Zolgensma was launched at a price of $2.1 million in the U.S., becoming the first drug to exceed $1 million. Drug manufacturers justify the high costs of these therapies with their potential to save lives in rare diseases.

The Swiss pharmaceutical company plans to present detailed results of the study at a medical meeting next year and discuss the findings with regulatory agencies. The drug was compared against a sham control, which is a procedure designed to mimic the administration of an experimental drug without providing active treatment.