Novartis has reported significant advancements in its investigational treatment for spinal muscular atrophy (SMA), as results from a late-stage trial have demonstrated promising outcomes. This genetic disorder is notable for the absence of a functional SMN1 gene, ultimately leading to the irreversible loss of motor neurons, which critically affects essential muscle functions, including breathing, swallowing, and movement.
Patients often rely on specialized care to enhance their functional capabilities. The pivotal phase 3 Steer trial was meticulously designed to assess the efficacy, safety, and tolerability of a single dose of the investigational gene therapy administered to treatment-naïve patients diagnosed with SMA Type 2.
The randomized, double-blind, and sham-controlled study successfully enrolled participants aged from two to under 18 years who have the capability to sit independently but have never been able to walk unaided. One of the most compelling findings from the trial was its achievement of the primary endpoint, highlighting substantial improvements in patients' motor functions within the first year.
This performance was quantitatively evaluated using the Hammersmith Functional Motor Scale - Expanded, ensuring robust and credible results. Additionally, the therapy displayed a favorable safety profile, with common side effects, such as upper respiratory tract infections, pyrexia, and vomiting, being reported similarly across all study arms. Shreeram Aradhye, president of development and chief medical officer at Novartis, commented, "The totality of evidence clearly supports a positive risk-benefit profile of OAV101, which we expect will facilitate registration to cover a broad spectrum of SMA patients.
Our commitment to innovating SMA treatment through our one-time gene therapies, specifically crafted to restore the function of the absent or defective SMN1 gene, remains strong." Following the successful outcomes from the Steer trial, Novartis, headquartered in Switzerland, has announced plans to submit the relevant data to regulatory authorities, including the US Food and Drug Administration (FDA), by 2025.
Furthermore, this data will be showcased at a significant medical event scheduled for that same year. As a reflection of the positive news regarding the trial results, shares in Novartis saw a modest increase during early trading hours..