AveXis’ Zolgensma gene therapy yields desired results in SMA trials

AveXis said that a one-time infusion of Zolgensma (onasemnogene abeparvovec-xioi) delivered rapid, substantial, and clinically meaningful therapeutic benefit in spinal muscular atrophy (SMA) patients across a range of trials.

Zolgensma also delivered the desired results in patients who were given presymptomatic treatment and also in certain participants aged over five years. The gene therapy could sustain durability as well in patients for up to five years post-dosing, said AveXis, which was acquired by Novartis in 2018 for $8.7bn.

Formerly known as AVXS-101, the gene therapy has approval from the US Food and Drug Administration (FDA) for the treatment of spinal muscular atrophy (SMA) in pediatric patients aged below two years of age, who have bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

According to Novartis, interim data from the SPR1NT trial showed presymptomatic babies with spinal muscular atrophy treated with the gene therapy soon after their birth could achieve age-appropriate motor milestones.

The Swiss pharma company said that apart from meeting the two co-primary efficacy endpoints, nine of 22 patients in the completed STR1VE-US trial showed the "ability to thrive", compared to untreated children having spinal muscular atrophy Type 1.

In the ongoing START long-term follow-up study, the gene therapy delivered sustained durability, which included the achievement and maintenance of milestones through the follow up period, which is now up to five years post-dosing and in certain patients aged over five years.

Novartis said that the cumulative safety data from 335 patients treated with the Zolgensma gene therapy indicate a safety profile on par with previously-reported safety information.

AveXis chief medical officer Olga Santiago said: "SMA is a disease that robs babies of the ability to talk, eat, sit up and even breathe. In complete contrast to the natural course of the disease, patients who received Zolgensma soon after birth before the onset of symptoms are achieving age-appropriate motor milestone development – an extraordinary outcome for SMA patients.

"These SPR1NT data demonstrate the truly transformational impact a one-time dose of gene therapy can have, and further underscore the importance of newborn screening and early intervention to alter the course of the disease."

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