Novartis has received a positive opinion from the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) for Zolgensma in spinal muscular atrophy (SMA), and Cosentyx in non-radiographic axial spondyloarthritis (nr-axSpA).
The opinion will be considered by the European Commission (EC) while reviewing the drugs for marketing approval.
Novartis subsidiary AveXis secured the CHMP recommendation for the use of Zolgensma (onasemnogene abeparvovec) to treat 5q SMA type 1 patients having a bi-allelic mutation in the SMN1 gene or 5q SMA patients with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene.
Designed as one-time gene therapy, the drug targets the genetic root cause of the disease, replacing the function of the missing or non-functioning SMN1 gene.
The CHMP opinion comes after the review of the completed Phase III STR1VE-US and Phase I START clinical trials performed to evaluate the safety and efficacy of one-time IV infusion of the drug in symptomatic SMA Type 1 patients aged under 6 months.
Furthermore, the decision supported interim data from the ongoing Phase III SPR1NT trial in presymptomatic patients.
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Furthermore, the drug is being studied in the Phase III STR1VE-EU trial.
The Japanese Ministry of Health, Labour and Welfare approved the drug earlier this month for SMA treatment in patients aged below two years, including those with a pre-symptomatic diagnosis.
CHMP recommended Novartis’ Cosentyx (secukinumab) to treat active non-radiographic axial spondyloarthritis (nr-axSpA) in adults.
The drug is a fully-human biologic that directly blocks IL-17A, a cytokine involved in the inflammation and development of PsO, PsA and AS.
CHMP’s opinion of Cosentyx comes from safety and efficacy outcomes from the Phase III PREVENT trial conducted in 555 participants.
The trial enrolled adult patients with objective signs of inflammation who did not have an adequate response to nonsteroidal anti-inflammatory drugs (NSAIDs).
Novartis Immunology, Hepatology & Dermatology global development unit head Eric Hughes said: “Non-radiographic axial spondyloarthritis is part of the axSpA spectrum and is a painful and debilitating disease for which there are limited treatment options available.
“This positive opinion marks another step forward in our commitment to reimagine medicine in axSpA and help patients realise relief from the burdensome symptoms of their disease earlier.”