Novartis' OAV101 IT Gene Therapy Shows Motor Function Improvement in Older SMA Patients

• Novartis' gene therapy, OAV101 IT (onasemnogene abeparvovec), has demonstrated improved motor function in older children (2-18 years) with spinal muscular atrophy (SMA) in a late-stage trial.
• The study included SMA Type 2 patients who could sit but never walk independently, showing improvements in sitting, rolling, crawling, and standing compared to a sham control.
• OAV101 IT, which shares Zolgensma's active ingredient, aims to extend Novartis' treatment options for older SMA patients, with plans to present detailed results and discuss with regulators.
• The therapy exhibited a favorable safety profile, with common side effects including upper respiratory tract infection, fever, and vomiting.

Novartis AG has announced positive results from a late-stage trial of its gene therapy, onasemnogene abeparvovec (OAV101 IT), in older children with spinal muscular atrophy (SMA). The study demonstrated improvements in motor function in patients aged 2 to under 18 years with SMA who were administered the therapy.

The trial focused on patients with SMA Type 2 who had the ability to sit but had not achieved independent walking. These patients received OAV101 IT, which is administered differently than the already approved Zolgensma and is designed to address the needs of older children with SMA.

Crystal Proud, principal investigator at Children's Hospital of the King's Daughters, emphasized the importance of maintaining motor function for older SMA patients, stating that it "allows them to perform daily activities as independently as possible." The study assessed improvements in motor skills such as sitting, rolling, crawling, and standing using a standardized scale. Results indicated that patients treated with OAV101 IT showed significant improvement in these measures compared to those who underwent a sham procedure.

Spinal muscular atrophy is a genetic disorder characterized by the deficiency or absence of a protein essential for neuromuscular development. This deficiency leads to muscle weakness and atrophy, affecting essential functions like walking, talking, and swallowing. Zolgensma, Novartis's existing gene therapy, is already approved in the US for children under two years of age with SMA. OAV101 IT shares Zolgensma's active ingredient but is tailored for older children.

The company plans to present detailed findings from the trial at an upcoming medical meeting and will engage in discussions with regulatory bodies regarding potential approval pathways. Novartis noted that OAV101 IT displayed a favorable safety profile during the trial, with the most commonly reported side effects being upper respiratory tract infection, fever, and vomiting.

Zolgensma, when introduced, was priced at $2.1 million in the US, marking it as one of the most expensive single-dose treatments available. The introduction of OAV101 IT could broaden Novartis's portfolio in the rare disease treatment space, offering a potentially life-saving therapy to a wider range of SMA patients.