Novartis gene therapy poised to expand SMA treatment landscape

Swiss pharma giant Novartis (NOVN: VX) has recently announced that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) successfully achieved the primary endpoint in a Phase III STEER study (NCT05089656) involving pediatric patients aged two–17 years with type II spinal muscular atrophy (SMA).

These positive results have the potential to broaden the eligible patient population for this gene transfer therapy, according to pharma analytics company GlobalData.

GlobalData’s report, “Spinal Muscular Atrophy: Opportunity Assessment and Forecast,” reveals that the combined sales of IT and intravenous (IV) formulations of onasemnogene abeparvovec are projected to increase from $660.6 million in 2023 to $763.8 million by 2033 in the seven major markets (7MM; USA, France, Germany, Italy, Spain, UK and Japan) due to the growing awareness of SMA and the implementation of newborn screening (NBS) across the markets, coupled with an increase the eligible population for gene transfer therapy.