Words by Jade Williams
Novartis’ Phase III STEER study has met its primary endpoint, demonstrating a significant improvement in motor function in children and young adults with spinal muscular atrophy (SMA).
The study focused on treatment-naïve patients aged two to under 18 years who are able to sit but have never walked independently. Key findings revealed that Novartis’ intrathecal onasemnogene abeparvovec (OAV101 IT) demonstrated a significant improvement in motor function, as measured by the Hammersmith Functional Motor Scale – Expanded. Patients treated in the study showed superior improvements in HFMSE scores compared to the control group, underscoring the potential of this one-time gene therapy.
Novartis aims to present the results to regulatory agencies, including the US FDA in 2025. The company is preparing for a broader rollout of the product, aiming to offer this transformative therapy to SMA patients worldwide. Additional data will be shared at a medical conference in 2025.
“Many patients with SMA currently rely on chronic treatments to manage their disease,” said Shreeram Aradhye, M.D., President, Development and Chief Medical Officer, Novartis in a press release. “We remain committed to leading innovation in SMA treatment through our one-time gene therapies, uniquely designed to replace the function of the missing or defective SMN1 gene.”
“Maintaining motor function is a key goal for many older patients with SMA,” said Crystal Proud, M.D., Pediatric Neurologist and a Principal Investigator at Children’s Hospital of the King’s Daughters. “This may allow them the capacity to continue to propel their electric wheelchair, feed themselves with intact hand to mouth function, and perform other activities of daily living as independently as possible.”
The STEER study results align with earlier findings from the Phase I/II STRONG study, which demonstrated meaningful increases in HFMSE scores over a year in younger SMA patients treated with OAV101 IT. This growing body of evidence further positions OAV101 IT as a potential treatment for SMA, offering hope for long-term benefits in motor function and independence and exemplifying the potential of gene therapies in rare diseases.
