FDA Approves Novartis Gene Therapy Itvisma for Spinal Muscular Atrophy
Novartis AG has received FDA approval for Itvisma, a gene replacement therapy for spinal muscular atrophy (SMA) in patients aged two and older. Itvisma is the first gene therapy targeting the genetic root cause of SMA, potentially reducing the need for chronic treatment. The approval follows positive Phase III clinical trials showing improved motor function. This marks a significant advancement in SMA treatment.
Novartis AG has received approval from the US Food and Drug Administration (FDA) for Itvisma® (onasemnogene abeparvovec-brve), making it the first and only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA) who have a confirmed mutation in the SMN1 gene. The one-time, fixed-dose treatment is designed to address the genetic root cause of SMA, potentially reducing the need for chronic therapy. The approval is based on positive results from Phase III clinical studies showing improved and stabilized motor function in patients, regardless of prior SMA treatment history. Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Novartis AG published the original content used to generate this news brief via EDGAR, the Electronic Data Gathering, Analysis, and Retrieval system operated by the U.S. Securities and Exchange Commission (Ref. ID: 0001171843-25-007547), on November 25, 2025, and is solely responsible for the information contained therein. © Copyright 2025 - Public Technologies (PUBT) Original Document: here
