NEW YORK – Novartis on Wednesday said it plans to submit marketing applications with regulatory agencies for OAV101 IT (onasemnogene abeparvovec), the company's intrathecal gene therapy for spinal muscular atrophy (SMA), in the first half of 2025 based on positive efficacy and safety data from the Phase III STEER trial.
An intravenous form of onasemnogene abeparvovec is already approved and sold in the US under the brand name Zolgensma for patients under 2 years of age. Basel, Switzerland-based Novartis has said it is seeking a broader indication for the gene therapy, which could be supported by data from the registrational STEER study. In this trial, Novartis is testing OAV101 in pediatric and young adult patients between the ages of 2 and 18 years old with intermediate SMA, who are able to sit but not walk independently.
The gene therapy is designed to deliver a functional copy of the SMN1 gene, mutations in which cause SMA, a rare genetic neuromuscular disorder.
"The data presented today from our OAV101 IT program reinforce our belief in this therapy, which has the potential to have a meaningful impact on a broad range of people with SMA through its continuous benefit via a one-time dose," Shreeram Aradhye, president of development and chief medical officer at Novartis, said in a statement.
In the STEER study, patients received either the gene therapy or a sham control. The study met its primary endpoint by showing that patients on the gene therapy had better Hammersmith Functional Motor Scale-Expanded (HFMSE) scores at 52 weeks than they did at baseline and that these improvements were greater for gene therapy-treated patients than those on the sham control.
The incidence of adverse events was similar between the treatment and sham control groups, according to Novartis. The most common adverse events in both arms were upper respiratory tract infection and pyrexia, and the most frequent serious adverse events among those in the gene therapy arm were pneumonia and vomiting.