Novartis's gene therapy, Zolgensma, is already approved in the U.S. for children under the age of two with spinal muscular atrophy (SMA), a severe genetic disorder that affects the muscles and is the leading genetic cause of infant mortality. Now, the treatment has the potential to expand its use to older children, following promising results from a late-stage trial. The trial focused on patients aged 2 to less than 18 who had never received prior treatment and were unable to walk, talk, or swallow due to the condition.
The therapy, onasemnogene abeparvovec (OAV101 IT) or Zolgensma, showed significant improvements in motor functions such as sitting, rolling, crawling, and standing in these older children. Compared to those who received a sham procedure, patients treated with OAV101 IT were able to perform basic daily activities more independently. The study enrolled over 100 patients with type 2 SMA, who could sit but had never walked. The results underscore the treatment's potential to address a critical unmet need in the SMA community.
Crystal Proud, principal investigator at Children's Hospital of the King's Daughters, emphasized that maintaining motor function is key for older SMA patients. The trial also demonstrated that OAV101 IT has a favorable safety profile, with the most common side effects being respiratory infections, fever, and vomiting. Novartis plans to present these results to regulatory agencies next year as part of the process to seek approval for the expanded use of the therapy in older children.
If approved, this new application of Zolgensma could offer a life-changing solution for children with SMA who are beyond the age threshold for the current treatment. This would position Novartis to further strengthen its leadership in gene therapy for rare diseases, competing with other SMA treatments such as Biogen’s Spinraza and Roche-PTC Therapeutics’ Evrysdi. Despite the news, Novartis shares remained steady, reflecting a long-term investor outlook as the company continues to make strides in gene therapy innovation.
Created: Jan 4th, 2025
Citations:
Reuters. (2024, December 30). Novartis gene therapy helps children with rare muscle disorder in study. Reuters.
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