The FDA has relaxed a clinical hold on clinical trials of Novartis' intrathecal formulation of spinal muscular atrophy (SMA) gene therapy Zolgensma, allowing a new phase 3 trial to get underway.

The intrathecal version comes at a time when Zolgensma sales have become stagnant, relying mainly on new geographic expansions. In the first nine months of 2024, sales from the one-time gene ...

Spinraza remains a big product for Biogen, but has seen its sales go into reverse as competition in SMA treatment has emerged ...

Novartis (NYSE:NVS) said a Phase 3 study of an intrathecal formulation of its gene therapy Zolgensma, also known as OAV101 IT, met its primary endpoint in the treatment of children with spinal ...

No new safety signals were observed in the study. In fiscal year 2023, Novartis reported Zolgensma sales of $1.21 billion . Zolgensma (onasemnogene abeparvovec) is the only approved gene therapy ...