Novartis Can Again Pursue Bigger Zolgensma Market After FDA Lifts OAV-101 Hold

Novartis AG is back on the path to potentially doubling the addressable market for its spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec) now that the US Food and Drug Administration has lifted a partial hold on the clinical trial for an intrathecal version of the product, known as OAV-101. While the move lifts an overhang from the program itself, the safety of adeno-associated virus (AAV)-based gene therapies in general remains an ongoing concern.

The Basel, Switzerland-based drug maker said on 3 August that the FDA lifted the partial clinical hold on its Phase I/II STRONG study after the company provided toxicology data from nonhuman primates (NHP) that addressed questions of dorsal root ganglia (DRG) injury after intrathecal administration