07:12 AM EST, 12/30/2024 (MT Newswires) -- Novartis ( NVS ) said in a regulatory filing Monday that its phase 3 study evaluating its one-time gene therapy intrathecal onasemnogene abeparvovec, or OAV101 IT, met its primary endpoint.

Based on the results, there was an increase from baseline in the Hammersmith Functional Motor Scale - Expanded, or HFMSE, total score in patients with spinal muscular atrophy treated with the drug candidate.

The study targeted patients aged two to less than 18 years who could sit but had never walked independently.

The phase 3 study demonstrated improvements in motor function as measured by the HFMSE scores, compared with a sham-controlled group.

The company said the safety profile was "favorable," with adverse events, such as respiratory tract infection, pyrexia and vomiting, similar between groups.

Novartis ( NVS ) said it plans to submit the findings to regulatory agencies, including the US Food and Drug Administration, next year.