Novartis: positive data in spinal muscular atrophy

(CercleFinance.com) - Novartis reports positive initial results from its pivotal Phase III STEER study, which evaluated the investigational intrathecal abepervovec onasemnogen (OAV101 IT) in patients with type 2 spinal muscular atrophy (SMA), aged two to under 18 years.


The study met its primary endpoint, showing improvement from baseline across the study population in Hammersmith Functional Motor Scale - Expanded (HFMSE) total scores.

OAV101 IT is the first investigational gene therapy to provide clinical benefit in treatment-naive AS patients aged two years and older with a positive risk-benefit profile, the Swiss healthcare group said.

This data will be presented at an upcoming medical meeting, and Novartis plans to share them with regulators in 2025, including the US FDA, with the aim of making OAV101 IT available to patients in need.


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