Novartis has reported interim data from the ongoing Phase III STR1VE-EU clinical trial of Zolgensma (onasemnogene abeparvovec) for paediatric patients with spinal muscular atrophy (SMA) type 1.
New findings showed that patients continued to experience a significant therapeutic benefit when treated with Zolgensma.
This therapeutic benefit included event-free survival, motor function improvement, and reaching motor milestones.
SMA is a genetic neuromuscular disorder that developed due to lack of a functional SMN1 gene that causes progressive and irreversible loss of motor neurons.
The rare disease affects muscle functions such as breathing, swallowing, and basic movement.
Zolgensma targets the genetic root cause of SMA by delivering a functional copy of the human SMN gene to stop disease progression.
Zolgensma obtained approval from the US Food and Drug Administration (FDA) in May last year.
STR1VE-EU investigates the efficacy and safety of a single, one-time IV infusion of Zolgensma in SMA type 1 patients aged under six months, with one or two copies of the SMN2 backup gene and bi-allelic SMN1 gene deletion or point mutations.
Interim data showed that 65.6% of patients administered with Zolgensma achieved developmental motor milestones not seen in the natural history of the disease, at a mean follow-up duration of 10.6 months.
A further 18.8% of patients could sit independently for ten seconds or more, which was the primary efficacy endpoint.
Furthermore, 66.7% of participants gained head control and 25% were able to roll from back to sides. One patient could stand, crawl and walk with assistance.
The company added that 66.7% of subjects were able to feed without support, which is a key indicator of disease progression stabilisation or halt.
Novartis Gene Therapies chief medical officer Shephard Mpofu said: “These strong interim results from the STR1VE-EU clinical trial continue to demonstrate consistent and significant therapeutic benefit in patients with SMA Type 1, the most common form of the disease, adding to the robust body of clinical evidence for Zolgensma.”
In April last year, Novartis unit AveXis reported positive interim results from the Phase III STR1VE trial of the drug in SMA type 1.