Novartis NVS obtained approval from the European Commission (EC) for Itvisma (onasemnogene abeparvovec) for the treatment of children two years and older, teens and adults living with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the survival motor neuron 1 (SMN1) gene.
The approval makes Itvisma the first and only gene replacement therapy authorized in the EU for a broad population of SMA patients.
The therapy is designed to target the underlying genetic cause of SMA through a one-time fixed dose that does not require adjustment based on age or body weight. A new working copy of the human SMN1 gene is delivered into a patient’s cells. By replacing the SMN1 gene, Itvisma aims to improve motor function and offers an alternative to therapies requiring ongoing administration.
SMA is a rare inherited neuromuscular disorder caused by mutations or the absence of the SMN1 gene that results in the progressive and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.
Year to date, shares of Novartis have gained 11.2% compared to the industry’s gain of 11.2%.
More on NVS’ Itvisma
The EC approval is based on data from the registrational STEER study, along with findings from the phase IIIb STRENGTH and phase I/II STRONG studies. In the STEER trial, Itvisma achieved a statistically significant 2.39-point improvement in the Hammersmith Functional Motor Scale Expanded (HFMSE), with benefits maintained through 52 weeks of follow-up. Results from the STEER and STRENGTH studies also demonstrated clinically meaningful benefits in both treatment-naïve and previously treated patients.
We note that an intravenous formulation of onasemnogene abeparvovec is marketed as Zolgensma in the United States, EU and other markets to treat babies and young children who have SMA with biallelic mutations in the SMN1 gene.
Itvisma, the intrathecal formulation, is also approved in the United States to treat adults and pediatric patients 2 years of age and older living with SMA with a confirmed mutation in the SMN1 gene.
Itvisma expands access to a one-time gene replacement therapy for older children, adolescents, and adults, potentially addressing longstanding unmet needs.
With the latest EC approval of Itvisma, Novartis can now offer gene replacement therapy options spanning different stages of SMA in Europe, from newborns to adults.
Novartis’ neuroscience portfolio includes Kesimpta (relapsing forms of multiple sclerosis) and Aimovig (migraine prevention).
Novartis AG Price, Consensus and EPS Surprise
Novartis AG price-consensus-eps-surprise-chart | Novartis AG Quote
NVS Looks to Strengthen Pipeline/Portfolio
2026 is a pivotal year for Novartis as it navigates the largest patent expiry in its history.
Novartis is now banking on key growth drivers — Kisqali, Kesimpta, Pluvicto, Scemblix and Leqvio — to support top-line growth.
NVS is also focusing on strategic acquisitions to strengthen its pipeline.
The company is all set to acquire SNV4818 from Synnovation Therapeutics, a pan-mutant selective PI3Kα inhibitor currently in phase I/II for HR+/HER2- breast cancer and other advanced solid tumors. The program aligns with Novartis’ breast cancer strategy and could be combined with CDK inhibitors and endocrine therapies.
Novartis agreed to acquire Excellergy, including Exl-111, a phase I anti-IgE antibody with extended half-life and high affinity. Its differentiated mechanism may enable faster and deeper receptor downregulation, potentially improving symptom control and dosing convenience in allergic diseases. The transaction is expected to be closed in the second half of 2026, subject to customary conditions.
NVS’ Zacks Rank & Stocks to Consider
Novartis currently carries a Zacks Rank #3 (Hold).
A couple of better-ranked biotech and large-cap pharma stocks are Immunocore IMCR and Bayer AG BAYRY. While IMCR sports a Zacks Rank #1 (Strong Buy), Bayer carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 30 days, earnings per share (EPS) estimates for Immunocore have remained unchanged at 6 cents for 2026 and 87 cents for 2027.
Immunocore’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, the average surprise being 46.66%.
Shares of Bayer have gained 28% over the past year. Estimates for BAYRY’s 2026 EPS have increased from $1.20 to $1.26 over the past 60 days.
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