New late-breaker data from the Phase III trial for Zolgensma, the world’s most expensive drug, has shown positive results.
Zolgensma demonstrated age-appropriate development when used presymptomatically and rapid, clinically meaningful efficacy in symptomatic children, even those with severe SMA at baseline.
The completed Phase III STR1VE-EU trial demonstrated rapid improvements in motor function following treatment with Zolgensma, and the majority of patients achieved motor milestones not observed in the natural history of SMA Type 1.
The Zolgensma data represent a significant contrast to the natural history of SMA Type 1, which leads to progressive and irreversible loss of motor function and if left untreated, often death or permanent ventilation by the age of two years.
100% of the children treated presymptomatically in the SPR1NT two-copy cohort achieved event-free survival, were independent of respiratory and nutritional support and met the primary endpoint of sitting independently for ≥30 seconds, including 11/14 (79 percent) who achieved this milestone within the World Health Organization (WHO) window of normal development.
A majority of patients went on to stand independently (11/14) and walk independently (9/14), most within the typical range of normal development.
Shephard Mpofu, Chief Medical Officer, Novartis Gene Therapies, said: “With more than 1,200 children now treated, these data presented at EAN further reinforce the life-changing benefit of a one-time treatment of Zolgensma.
“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.”
Professor Eugenio Mercuri, Department of Paediatric Neurology, Catholic University, Italy, said: “STR1VE-EU included some patients with more severe SMA at baseline, yet the study demonstrated consistent and significant therapeutic benefit for symptomatic children with SMA Type 1.
“This is a remarkable outcome that adds to the robust body of clinical evidence for Zolgensma showing that even among patients with more severe disease, Zolgensma was highly effective and demonstrated a consistent safety profile.”
Among symptomatic children with SMA Type 1 treated in the STR1VE-EU trial, including patients with more severe disease at baseline, the majority of children (82 percent) achieved developmental motor milestones not observed in the natural history of SMA Type 1, including 16 children (49 percent) who sat without support for ≥30 seconds.
SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.
If left untreated, children with SMA have a life expectancy of two years.
Lilly Subbotin
This is a syndicated feed from Pharmafile
