Novartis AG (NYSE: NVS) announced that Itvisma (onasemnogene abeparvovec‑brve) received U.S. FDA approval for children aged two and older, teens, and adults with Spinal Muscular Atrophy (SMA) caused by SMN1 gene mutations, becoming the first and only gene replacement therapy for this broad population.
Regulatory Milestone
Item
Detail
Product
Itvisma (onasemnogene abeparvovec‑brve)
Company
Novartis AG (NYSE: NVS)
Agency
U.S. Food and Drug Administration (FDA)
Approval Date
24 Nov 2025
Indication
SMA in patients aged ≥2 years with confirmed SMN1 mutation
Significance
First gene replacement therapy for this population
Administration
Single one-time intrathecal injection
Drug Profile
Mechanism: Delivers functional copy of human SMN1 gene via adeno-associated viral vector
Innovation: Addresses genetic root cause of SMA rather than symptoms
Differentiation: First gene therapy approved for pediatric, adolescent, and adult SMA patients
Dosing: Single intrathecal injection for sustained SMN protein expression
Clinical Evidence
Study
Design
Key Findings
STEER
Phase III registrational
Statistically significant motor function improvements
STRENGTH
Phase IIIb open-label
Motor ability stabilization sustained over 52 weeks
Pipeline Expansion: Platform applicable to other neurological monogenic disorders
Forward-Looking Statements This brief contains forward-looking statements regarding Itvisma’s commercial performance, market penetration, and manufacturing capacity. Actual results may differ materially due to risks including pricing negotiations, competitive responses, and real-world efficacy data.-Fineline Info & Tech
By Fineline Cube 