Novartis Reveals Promising Gene Therapy Data for Treating Spinal Muscular Atrophy in Older Children
Portfolio Pulse from Vandana Singh
Novartis AG (NYSE:NVS) presented promising data from the SMART Phase 3b study for Zolgensma, a gene therapy for spinal muscular atrophy (SMA) in older children. The study showed safety and efficacy in children weighing ≥ 8.5 kg to ≤ 21 kg, with significant improvements in motor milestones and no new safety signals. Zolgensma sales reached $1.21 billion in fiscal year 2023. The therapy is approved in over 51 countries and has treated over 3,700 patients globally.

March 04, 2024 | 4:34 pm
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Novartis AG presented positive data for Zolgensma in treating SMA in older children, showing safety, efficacy, and significant sales figures.
The positive data from the SMART Phase 3b study for Zolgensma not only reinforces the therapy's safety and efficacy profile but also highlights its commercial success with $1.21 billion in sales. This could lead to increased investor confidence in Novartis's pipeline and financial health, potentially driving up the stock price in the short term.
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