US EditionMuscular Dystrophy Association Calls FDA Approval of Novartis’ Itvisma (Onasemnogene ...
Itvisma, a one-time gene therapy, showed sustained motor function improvements in Phase III trials and will be available in the US in December, Novartis said.
- Yesterday, Novartis said the U.S. Food and Drug Administration approved Itvisma for children two years and older, teens and adults with SMA, with US availability in December.
- Novartis based its submission on the Phase III STEER study and Phase IIIb STRENGTH study , showing motor improvements sustained over 52 weeks with a consistent safety profile.
- Designed as a one-time intrathecal administration, Itvisma delivers a functional SMN1 gene without dose adjustments, and John W. Day, MD, PhD, called its FDA approval a "game-changing advance".
- To assist patients, Novartis says the FDA approval helps address unmet needs among older children, teens, and adults and offers Novartis Patient Support; call 1-855-441-4363 for assistance.
- Novartis cautioned there is no guarantee Itvisma will be approved in other markets and stressed commercial success is not assured, noting development used exclusive licenses with Nationwide Children's Hospital, REGENXBIO, and Généthon.
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Muscular Dystrophy Association Calls FDA Approval of Novartis’ Itvisma (onasemnogene ...
New York, Nov. 24, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today called the U.S. Food and Drug Administration’s approval of Itvisma (onasemnogene abeparvovec-brve), developed by Novartis, a major step forward for families living with spinal muscular atrophy…
Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA)
Itvisma (onasemnogene abeparvovec-brve) demonstrated improved motor function and stabilization in patients regardless of SMA treatment history in Phase III studiesOne-time dose of Itvisma replaces SMN1 gene, potentially reducing the need for chronic SMA treatmentGene replacement therapy now available to eligible…
Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA)
Itvisma (onasemnogene abeparvovec-brve) demonstrated improved motor function and stabilization in patients regardless of SMA treatment history in Phase III studiesOne-time dose of Itvisma replaces SMN1 gene, potentially reducing the need for chronic SMA treatmentGene replacement therapy now available to eligible…
Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA)
Itvisma (onasemnogene abeparvovec-brve) demonstrated improved motor function and stabilization in patients regardless of SMA treatment history in Phase III studiesOne-time dose of Itvisma replaces SMN1 gene, potentially reducing the need for chronic SMA treatmentGene replacement therapy now available to eligible…
Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA)
Itvisma (onasemnogene abeparvovec-brve) demonstrated improved motor function and stabilization in patients regardless of SMA treatment history in Phase III studiesOne-time dose of Itvisma replaces SMN1 gene, potentially reducing the need for chronic SMA treatmentGene replacement therapy now available to eligible…
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