Core Insights - Novartis (NVS) received FDA approval for Itvisma, a gene replacement therapy for spinal muscular atrophy (SMA) in patients aged two years and older, including teens and adults [1][7] - Itvisma is the first and only gene replacement therapy targeting the genetic cause of SMA, delivering a functional SMN1 gene in a single fixed dose [2][3] FDA Approval and Clinical Data - The approval was based on positive results from the phase III STEER study and the open-label phase IIIb STRENGTH study, showing significant motor function gains and stabilization of motor abilities over 52 weeks [4][7] - Itvisma demonstrated a consistent safety profile across both studies, addressing the unmet needs of approximately 9,000 individuals in the U.S. affected by SMA [5] Company Strategy and Market Position - Novartis is focusing on diversifying its portfolio amid generic competition for key drugs like Entresto, with new drug approvals and label expansions expected to mitigate adverse impacts [6] - The company’s shares have increased by 30.1% year-to-date, outperforming the industry growth of 16% [6] Future Growth Projections - Management projects a compounded annual growth rate (CAGR) of 5-6% for sales from 2025 to 2030, with an upgraded sales guidance for 2024-2029 to 6% from 5% [9] - Strong performance from key products such as Kisqali, Kesimpta, Pluvicto, and Scemblix supports the company's growth despite challenges from generic competition [10] Recent Acquisitions - Novartis announced plans to acquire Avidity Biosciences for $12 billion to enhance its late-stage neuroscience pipeline, with the acquisition expected to close in the first half of 2026 [11][12]