Positive news for Novartis as Phase III study of intrathecal onasemnogene abeparvovec in SMA patients is successful.
From GlobeNewswire: 2024-12-30 00:45:00
Novartis announced positive results from the Phase III STEER study for intrathecal onasemnogene abeparvovec (OAV101 IT) in SMA patients aged two and older. The study met its primary endpoint by increasing HFMSE total scores compared to the sham control. The safety profile of OAV101 IT was favorable, with similar adverse events between groups. Novartis plans to share results with regulatory agencies in 2025 to make OAV101 IT available for SMA patients. These results show the efficacy, safety, and tolerability of OAV101 IT, supporting its potential registration for a broad range of SMA patients. A new Phase III study, STEER, showed that intrathecal onasemnogene abeparvovec (OAV101 IT) maintained and even increased motor function in SMA patients. The study included over 100 treatment-naïve patients with SMA Type 2, aged two to less than 18 years. OAV101 IT led to clinically meaningful increases in HFMSE scores over one year. The one-time gene therapy is designed to replace the function of the missing or defective SMN1 gene. Results from STEER build on previous studies showing the potential impact of OAV101 IT on SMA treatment. The therapy aims to help patients maintain independence in daily activities. Novartis has been conducting a clinical development program for the OAV101 IT gene therapy in approximately 170 patients with Spinal Muscular Atrophy (SMA) for up to 6.4 years. The company holds exclusive licenses for the delivery of the gene therapy from Nationwide Children’s Hospital, REGENXBIO, and Généthon. SMA is a rare genetic neuromuscular disease caused by a lack of a functional gene, leading to irreversible loss of motor neurons and affecting muscle functions. Novartis emphasizes that forward-looking statements in the press release are subject to known and unknown risks and uncertainties. Novartis is dedicated to reimagining medicine to improve and extend patients’ lives.
Read more at GlobeNewswire: Novartis intrathecal onasemnogene abeparvovec Phase III