FDA Greenlights Novartis' Gene Replacement Treatment for SMA

Novartis NVS obtained FDA approval for onasemnogene abeparvovec-brve, a gene replacement therapy, for children two years and older, teens, and adults with spinal muscular atrophy (SMA).

The therapy was approved under the brand name Itvisma for SMA patients with a confirmed mutation in the survival motor neuron 1 (SMN1) gene.

SMA is a rare genetic neuromuscular disorder caused by a mutation or missing SMN1 gene. The SMN1 gene produces a major portion of the survival motor neuron protein, which is essential for muscle function, including breathing, swallowing and everyday movements.

Per NVS, Itvisma is the first and only gene replacement therapy available for this broad population.

The therapy is specifically designed to target the genetic root cause of SMA with a one-time, fixed dose that requires no adjustment for age or body weight. By delivering a functional SMN1 gene, Itvisma can enhance motor function and may reduce the need for ongoing, chronically administered therapies.

More on NVS’ Gene Replacement Therapy for SMA

The FDA approval of Itvisma was based on positive data from the registrational phase lll STEER study and supported by the open-label phase lllb STRENGTH study.

Study results showed that Itvisma produced statistically significant gains in motor function and stabilized motor abilities — outcomes not typically observed in the natural progression of SMA. These benefits were maintained through 52 weeks of follow-up.

Itvisma also demonstrated a consistent safety profile, with adverse consistent aligned across both studies.

An estimated 9,000 individuals in the United States are affected by SMA. Despite therapeutic progress, unmet needs remain, especially among older children, teenagers, and adults seeking to preserve motor neurons and sustain functional strength.

NVS Focuses on Broadening Portfolio Amid Generic Competition

Novartis’ efforts to strengthen and diversify its broad portfolio as key drugs like Entresto face generic competition are impressive.

Approval of new drugs and label expansion of existing drugs should enable the company to offset the adverse impacts of generic competition for Tasigna, Promacta and Entresto.

Shares of Novartis have gained 30.1% year to date compared with the industry’s growth of 16%.

NVS is currently focused on four core therapeutic areas — cardiovascular-renal-metabolic, immunology, neuroscience and oncology.

Novartis recently received FDA approval for remibrutinib, under the brand name Rhapsido, as an oral treatment for adult patients with chronic spontaneous urticaria (CSU) who remain symptomatic despite H1 antihistamine treatment. The approval makes it the first FDA-approved Bruton’s tyrosine kinase inhibitor (BTKi) for CSU.

Last week, management projected sales to witness a compounded annual growth rate (CAGR) of 5-6% (at constant currencies) for 2025-2030.

Management also upgraded its sales guidance for 2024-2029 to 6% from 5% (projected earlier).

The upgrade can be attributed to continued strong momentum from in-market growth drivers and upcoming launches.

While generic competition for its blockbuster drug Entresto in the United States poses a major headwind, strong performance from key products such as Kisqali, Kesimpta, Pluvicto, and Scemblix continues to support the company’s momentum.

Novartis recently announced that it will acquire San Diego-based Avidity Biosciences, Inc RNA for $12 billion to strengthen its late-stage neuroscience pipeline. As part of the agreement, Avidity will spin off its early-stage precision cardiology programs into a new company before the closing of the acquisition.

Avidity is developing RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs) for serious, genetic neuromuscular diseases.

This acquisition is scheduled to be closed in the first half of 2026, subject to the separation of cardiology programs from Avidity and other customary closing conditions.

NVS’ Zacks Rank and Stocks to Consider

Novartis currently carries a Zacks Rank #3 (Hold). A couple of better-ranked pharma and biotech stocks are Amicus Therapeutics FOLD and Bayer BAYRY. While FOLD currently 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.

In the past 60 days, estimates for Amicus Therapeutics’ 2025 EPS have increased to 34 cents from 31 cents. During the same time, EPS estimates for 2026 have decreased to 67 cents from 69 cents. Year to date, shares of FOLD have gained 5%.

Bayer’s shares have risen 79.1% so far this year. Estimates for 2025 EPS have increased from $1.37 to $1.40 over the past 60 days, while those for 2026 EPS have risen from $1.44 to $1.45. 

 

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