The approval allows Zolgensma to treat patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene.
As per the approved dosing guidance, the Zolgensma has to be used for the treatment of babies and young children with SMA up to 21kg.
According to the company, between 550 and 600 infants are born with SMA, which is a rare and genetic neuromuscular disease caused by a lack of a functional SMN1 gene.
The approval was based on data from the phase 3 STR1VE-US and phase 1 START trials, which assessed the efficacy and safety of a one-time IV infusion of Zolgensma in symptomatic SMA Type 1 patients less than six months of age at dosing, who had one or two copies of the SMN2 backup gene, or two copies of the SMN2 backup gene, respectively.
The comparable and ongoing phase 3 STR1VE-EU trial showed prolonged event-free survival and rapid motor function improvement, said the company.
Zolgensma has been designed to offer a functional copy of the human SMN gene to restrict SMA disease progression via sustained SMN protein expression with a single, one-time IV infusion.
The company has also announced Day One access programme for Zolgensma, which helps to maintain the integrity of the local pricing and reimbursement frameworks with different customisable options.
In May 2019, the company secured approval from the US Food and Drug Administration for Zolgensma to treat pediatric patients less than two years of age with SMA with bi-allelic mutations in the SMN1 gene.
IN March this year, the firm received approval from the Ministry of Health, Labour and Welfare (MHLW) for Zolgensma to treat SMA in patients under the age of two, including those who are pre-symptomatic at diagnosis.
AveXis president Dave Lennon said: “The EC approval of Zolgensma is a significant milestone for the SMA community, and further underscores the substantial clinical value of the only gene therapy for SMA, bringing new hope to those impacted by this rare, but devastating disease.
“Even under the current pandemic conditions, the urgent need to treat SMA has resulted in access pathways in France and Germany for Zolgensma, a potentially life-saving medicine delivered in a single dose.”
Earlier this month, Novartis secured approval from the FDA for its Tabrecta (capmatinib) to treat adult patients metastatic non-small cell lung cancer (NSCLC) with MET exon 14 skipping (METex14).