Novartis Reports Data from P-III (STEER) Study of Onasemnogene Abeparvovec to Treat Children and Young Adults with Spinal Muscular Atrophy (SMA)
Shots:
- The sham-controlled P-III (STEER) trial assessed safety, efficacy & tolerability of onasemnogene abeparvovec (OAV101 IT) in treatment-naïve SMA Type 2 patients (n= >100; age: 2–18yrs.) who can sit but have never walked. Eligible patients switched treatments after 52wks.
- The study achieved its 1EP, depicting an increase in total Hammersmith Functional Motor Scale - Expanded (HFMSE) scores from baseline, suggesting better motor function in patients
- Safety profile was favorable showing similar AEs, with common ones being upper respiratory infection, fever & vomiting. Novartis will submit results to regulatory agencies in 2025 and highlight them at future conferences
Ref: Novartis | Image: Novartis
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
