Swiss pharmaceutical giant Novartis (NYSE: NVS) has announced positive topline results from the Phase III STEER study, which was designed to assess the efficacy and safety of its intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve patients with spinal muscular atrophy (SMA) Type 2. The study focused on patients aged two to less than 18 years who are able to sit but have never walked independently.
OAV101 IT: An Adeno-Associated Virus (AAV) Gene Therapy
Onasemnogene abeparvovec, an adeno-associated virus (AAV) gene therapy, has seen its intravenous infusion dosage form, Zolgensma, approved in the United States in May 2019. The Phase III STEER study for OAV101 IT has successfully reached its primary endpoint, demonstrating an increase from baseline in the Hammersmith Functional Motor Scale – Expanded (HFMSE) total score, a key measure of motor function in SMA patients.
Sharing Results with Regulatory Agencies
The positive results from the STEER study are expected to be shared with regulatory agencies in 2025, including the US Food and Drug Administration (FDA). This sharing of data is a crucial step towards potentially expanding the treatment options for SMA Type 2 patients and further establishing gene therapy as a viable approach for addressing this debilitating condition.-Fineline Info & Tech
