Following the FDA, the European Commission has also approved Itvisma (onasemnogene abeparvovec) for the treatment of Spinal Muscular Atrophy (SMA). Created on August 16, 2026. Last updated:

Itvisma replaces defective gene responsible for spinal muscular atrophy

Novartis has secured EU approval for Itvisma, a new formulation of its spinal muscular atrophy gene therapy usable in a broader patient population.

FDA Approves Gene Therapy for Treatment of Spinal Muscular Atrophy

Novartis announced the FDA approval of Itvisma, a novel gene replacement therapy designed for individuals living with spinal muscular atrophy (SMA) aged two

Novartis today announced  that it has received approval from the U.S. Food and Drug Administration (FDA) for Itvisma (onasemnogene abeparvovec-brve) to treat adults and children 2 years of age and old...

Novartis has secured EU approval for Itvisma, a new formulation of its spinal muscular atrophy gene therapy usable in a broader patient population.

Die US-Arzneimittelbehörde FDA hat das Gentherapie-Medikament Itvisma® (onasemnogene abeparvovec-brve) von Novartis für die Behandlung von Kindern ab zwei Jahren, Jugendlichen und Erwachsenen mit spinaler Muskelatrophie (SMA) zugelassen – sofern eine Mutation im SMN1-Gen bestätigt ist. Damit steht erstmals eine Einmal-Gentherapie für diese breite Altersgruppe zur Verfügung, die die genetische Ursache der Erkrankung direkt behebt und potenziell den Bedarf an chronischer Medikation reduziert.

YouTube video by Progress, Potential, and Possibilities

Sarepta hits more roadblocks while Novartis’ high-priced SMA gene therapy finds a new route to patients.

The FDA granted full approval for Novartis' Fabhalta to slow kidney function decline in adults with primary IgA nephropathy who are at risk of progression. This traditional approval represents a significant regulatory milestone for the treatment of this progressive kidney disease, expanding Novartis' portfolio of high-innovation medicines and supporting its strategic shift toward advanced therapies.

This edition highlights major developments across gene and cell therapy, including FDA approval of TREGZI, China's approval of satri-cel, progress toward the RGX-202 BLA submission, initiation of the Phase IIb/III NAAVIGATE study for sura-vec, expanded eligibility for CASGEVY, European approval of Itvisma for spinal muscular atrophy, a new Korean cell and gene therapy alliance led by GC Cell, and MeiraGTx's financing to support commercialization of late-stage gene therapy programs.

This biweekly Gene and Cell Therapy video recap highlights regulatory milestones, clinical progress, manufacturing collaborations, financing activity, and advancing development across multiple therapeutic areas. Key highlights include: 🧬 RGX-202 BLA path advances 👁️ Sura-vec NAAVIGATE begins 🧫 Satri-cel wins China approval 🩸 TREGZI gains FDA approval 👶 CASGEVY label expands to age 2+ 🧬 Itvisma wins EU SMA approval 🤝 GC Cell forms Korean CGT alliance 💰 MeiraGTx secures Oberland financing Chapters: 0:00 Introduction 0:09 RGX-202 BLA path advances 0:35 Sura-vec NAAVIGATE begins 0:58 Satri-cel wins China approval 1:18 TREGZI gains FDA approval 1:42 CASGEVY label expands to age 2+ 2:04 Itvisma wins EU SMA approval 2:24 GC Cell forms Korean CGT alliance 2:46 MeiraGTx secures Oberland financing 3:08 How to reach us 📢 Stay Ahead in Gene and Cell Therapy Research! ✅ Like, share, and subscribe for future updates on Gene and Cell Therapy ✅ Visit www.lqventures.com for expert healthcare insights and consulting services. ✅ Contact LucidQuest at info@lqventures.com for strategic guidance on Gene and Cell Therapy innovations and clinical research. 🔔 Turn on notifications so you never miss an update! #GeneandCellTherapy #GeneTherapy #CellTherapy #FDA #ClinicalTrials #CART #RareDisease #Biotech #PharmaNews #LucidQuest