Novartis reports phase 3 win for gene therapy in SMA

Novartis has revealed positive phase 3 trial results for its investigational gene therapy, onasemnogene abeparvovec, in children and young adults with spinal muscular atrophy (SMA) Type 2.

The STEER study evaluated the clinical efficacy, safety and tolerability of a one-time dose of intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment naïve patients with SMA Type 2, aged two to less than 18 years who were able to sit, but had never walked independently. According to Novartis, the study met its primary endpoint showing an increase from baseline across the study population in total Hammersmith Functional Motor Scale - Expanded (HFMSE) scores, considered the gold standard for SMA-specific assessment of motor ability and disease progression.

In terms of safety, the overall adverse events and serious adverse events were similar between the treatment arm and the sham control arm. The most common adverse events were upper respiratory tract infection, pyrexia and vomiting.

The new treatment shares the same active ingredient as Novartis’ blockbuster SMA treatment, Zolgensma, approved in 2019. Zolgensma is approved for children less than two years old and is administered intravenously, while OAV101 IT targets older children and is administered intrathecally (through the spinal column).

The recent STEER results make OAV101 IT the first investigational gene therapy to provide clinical benefit in treatment-naïve patients with SMA aged two and older with a positive risk benefit profile. Novartis says it plans to share results with regulatory agencies in 2025.