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Novartis publishes final positive phase 3 results of Zolgensma in Nature Medicine

Almost all children involved with copies of the SMN2 gene treated pre-symptomatically met key milestones


Final results from Novartis’ phase 3 SPR1NT trial assessing Zolgensma (onasemnogene abeparvovec) have been published in Nature Medicine. The results showed that almost all children with two or three copies of the SMN2 gene treated pre-symptomatically had met key milestones including sitting, walking and standing.

The results were taken from both the two- and three-copy groups of the phase 3 study as separate companion manuscripts and further support the benefits of Zolgensma when administered early to children with spinal muscular atrophy (SMA).

SMA is a disease affecting an estimated one in 10,000 infants globally. If the condition is left untreated, patients with two copies of the SMN2 gene will typically go on to develop SMA type 1,  leading to a progressive and irreversible loss of motor function and, in the majority of cases, death or permanent ventilation by the age of two.

Most children diagnosed with three copies of the SMN2 gene develop SMA type 2, distinguished by the inability to walk independently.

The SPR1NT data showed that 100% of children treated pre-symptomatically in the two-copy group met the primary endpoint of sitting independently for more than 30 seconds, including 79% who achieved this milestone according to the World Health Organization (WHO) time frame of regular development.

In the three-copy group of SPR1NT, 93% of children went on to walk independently, while 73% were within the WHO window of normal development.

Zolgensma is currently the only approved gene therapy for the treatment of SMA and is the only SMA treatment produced to directly tackle the genetic root cause of the disease.

This is done by replacing the function of the missing or dysfunctional SMN1 gene to stop disease progression through sustained SMN protein expression via a single, one-off intravenous infusion.

“The robust data from both the two- and three-copy SPR1NT cohorts are being published together for the first time, further supporting the significant and clinically meaningful benefit of Zolgensma in presymptomatic babies with SMA,” said Shephard Mpofu, senior vice president, chief medical officer, Novartis Gene Therapies.

“When treated with Zolgensma prior to the onset of symptoms, not only did all 29 patients enrolled in SPR1NT survive but they were thriving – breathing and eating on their own, with most even sitting, standing and walking without assistance.”

Article by
Fleur Jeffries

20th June 2022

From: Research, Healthcare



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