New data for Novartis’ gene therapy Zolgensma has shown benefit in presymptomatic spinal muscular atrophy patients, the Swiss pharma company revealed at the European Academy for Neurology (EAN) virtual congress.
Zolgensma was initially approved in 2019 for the treatment of SMA, launching with a list price of $2.125m, making it the most expensive medicine in the world.
Since then, Zolgensma has been in competition with Biogen’s SMA drug Spinraza, approved in 2016, and Roche’s newly approved oral therapy Evrysdi.
In a boost to its clinical profile in SMA, Novartis has touted data from the phase 3 SPR1NT trial, in which Zolgensma hit the primary and secondary endpoints of helping patients sit independently for 30 seconds and reach 14 months of age without the need for ventilatory support.
In the open-label study, 14 patients with two copies of the SMN2 gene and 15 patients with three copies of SMN2 were treated with Zolgensma.
In these presymptomatic patients, Zolgensma also helped patients to stand and walk independently, with all patients being independent of nutritional and respiratory support for the duration of the study.
Results from another study – STR1VE – were also presented at the EAN virtual congress. This trial evaluated Zolgensma in patients six weeks or younger with SMA type 1 with biallelic SMN1 gene deletion or point mutations and one or two copies of SMN2.
In this study, 82% of patients achieved development motor milestones not observed in the natural history of the disease.
In addition, 44% of patients in the intention-to-treat (ITT) population achieved the primary endpoint of sitting independently for ≥10 seconds.
“With more than 1,200 children now treated, this data presented at EAN further reinforces the life-changing benefit of a one-time treatment of Zolgensma,” said Shephard Mpofu, chief medical officer, Novartis Gene Therapies.
“When treated with Zolgensma prior to the onset of symptoms, not only did all patients survive, but were thriving — breathing and eating on their own and sitting independently, with many standing and walking.
“When you consider these newborns would go on to develop severe symptoms of SMA type 1, a devastating, progressive disease that robs children of the ability to talk, eat, sit up and even breathe, findings from the SPR1NT trial are nothing short of extraordinary,” he added.