Novartis subsidiary AveXis has received approval from the US Food and Drug Administration (FDA) for Zolgensma to treat children with spinal muscular atrophy (SMA).
The gene therapy drug has been designed for patients under the age of two that have bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
SMA is a rare, genetic neuromuscular disorder that develops due to a defective or missing SMN1 gene. It can lead to loss of the motor neurons associated with muscle functions such as breathing, swallowing, speaking and walking.
In its most severe form, the condition leads to paralysis, permanent ventilation or death by age two in more than 90% of patients.
Zolgensma is intended to address the genetic root cause of SMA by delivering a functional copy of the human SMN gene, aiming to halt progression of the disease through sustained expression of the SMN protein.
Novartis CEO Vas Narasimhan said: “The approval of Zolgensma is a testament to the transformational impact gene therapies can have in reimagining the treatment of life-threatening genetic diseases like spinal muscular atrophy.
“We believe Zolgensma could create a lifetime of possibilities for the children and families impacted by this devastating condition.”
The FDA decision is based on results from the ongoing Phase III STR1VE trial and the completed Phase I START trial of a one-time intravenous Zolgensma in SMA Type 1 patients.
Novartis said that the trial data showed significant survival rates, rapid motor function improvement and sustained achievement of milestones such as the ability to sit without support.
The safety profile of the drug was found to be comparable between trials.
The most common adverse effects were increased aminotransferases and vomiting.
Zolgensma will be marketed in the US by AveXis. The drug has a list price of $2.125m for a one-time therapy.
Separately, the FDA has approved Novartis’ Piqray (alpelisib) in combination with hormone therapy drug Fulvestrant to treat breast cancer in men and postmenopausal women.