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Novartis Gets EC Approval for Gene Therapy Zolgensma in SMA

Zacks Equity Research

Novartis NVS announced that the European Commission (EC) granted conditional approval to gene therapy, Zolgensma (onasemnogeneabeparvovec), for spinal muscular atrophy (SMA).

We note that Zolgensma is a one-time gene therapy designed to address the genetic root cause of the disease by replacing the function of the missing or nonworking SMN1 gene.

Zolgensma has been approved 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. The approval covers babies and young children with SMA (up to 21 kg), according to the approved dosing guidance.

SMA is a rare, genetic neuromuscular disease caused by the lack of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movements.

Zolgensma delivers a new working copy of the SMN1 gene into a patient’s cells, thereby halting disease progression following a single, intravenous (IV) infusion.
Zolgensma has demonstrated significant and clinically meaningful therapeutic benefit in pre-symptomatic and symptomatic SMA, including prolonged event-free survival and achievement of motor milestones.

It was added to Novartis’ portfolio through the acquisition of AveXis.

AveXis is in discussions with EU governments and reimbursement agencies to agree on terms of the innovative “Day One” access program to enable rapid access in all EU countries, given the urgent need to treat SMA.

Last year, the FDA approved Zolgensma for the treatment of pediatric patients less than two years of age with SMA, with bi-allelic mutations in the SMN1 gene. It was also approved in Japan in March 2020.

Other SMA treatments include Biogen’s BIIB Spinraza. Roche RHHBY is also evaluating risdiplam for SMA.

The stock has lost 10.4% in the year so far compared with the industry’s decline of 0.8%.


Last month, Novartis reported strong first-quarter results, driven by the solid performance of key drugs like Cosentyx and Entresto, and contribution from Zolgensma. COVID-19-related forward purchasing also boosted performance. New launches like Piqray and Beovu should boost the company’s performance in the upcoming quarters. In particular, strong uptake is expected from Beovu as it is the first FDA-approved anti-VEGF to offer greater fluid resolution as compared to Regeneron and Bayer’s BAYRY market-leading drug, Eylea.

Novartis currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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