Novartis Gene Therapies has unveiled new interim data from the ongoing Phase III STR1VE-EU clinical trial showing the benefit of Zolgensma (onasemnogene abeparvovec) in patients with spinal muscular atrophy (SMA) Type 1.
The group said patients taking the therapy “continued to experience significant therapeutic benefit, including event-free survival, rapid and sustained improvement in motor function and motor milestone achievement, including for some patients with more aggressive disease at baseline compared to previous trials”.
SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene that causes progressive and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.
Zolgensma targets the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion.
STR1VE-EU is designed to evaluate the efficacy and safety of a single, one-time IV infusion of Zolgensma in patients with SMA Type 1 who are less than six months of age at the time of gene therapy, with one or two copies of the SMN2 backup gene and who have bi-allelic SMN1 gene deletion or point mutations.
The interim data showed that 65.6% of SMA patients given Zolgensma achieved motor milestones not observed in the natural history of SMA Type 1, including: 18.8% who could sit independently for ≥10 seconds (the primary efficacy endpoint); 66.7% who gained head control; 25% who were able to roll from back to sides; and one patient who could stand with assistance, crawl and walk with assistance.
Also, 66.7% of patients in the intent-to-treat population were able to feed orally without the need for feeding support, an important indicator of stabilisation/halting of disease progression, the firm noted.
“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,” said Shephard Mpofu, chief medical officer, Novartis Gene Therapies.
“With more than 600 patients now treated, including some more than five years post-treatment and more than five years old, these data further reinforce the transformative benefit a one-time dose of Zolgensma has on SMA patients.”
The European Commission granted conditional approval for Zolgensma in May this year, allowing its use for the treatment of patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1; or for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene.
