Novartis appears happy so far with the performance of its drug Zolgensma (onasemnogene abeparvovec), a one-time gene replacement therapy for the treatment of spinal muscular atrophy (SMA).
The company presented results from a clinical study, called the SMART study (NCT04851873), of the gene therapy for previously treated patients and a broader patient population older and heavier (1.5 to 9.1 years old) than those treated in previous clinical studies. SMART was a phase IIIB, open-label, single-arm, multicenter study.
"The new clinical results supplement emerging real-world experience and use of this innovative therapy in older and heavier children in countries where authorized use is not restricted by age," Novartis said in a news release highlighting its presentation at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Orlando, FL.
"Most patients in the SMART study maintained motor milestones observed at baseline throughout the one-year study," Novartis said. Among the results, three patients achieved the milestone of newly standing with support and one achieved newly walking with support at Week 52. Most patients maintained or improved motor milestones from baseline at the time of final analysis. There were no reported cases of acute liver failure or bilirubin elevations, according to the company.
