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Novartis Hopes To Broaden SMA Patient Pool After Intrathecal Zolgensma Success

 
• By Kevin Grogan

The Swiss giant’s blockbuster gene therapy is currently only administered intravenously into patients with spinal muscular atrophy under two years of age. Its intrathecal candidate, called OAV-101, has just passed a Phase III test which could expand its use in children and young adults.

Surgical doctor looking at radiological spinal x-ray film for medical diagnosis on patient’s health on spine disease, bone cancer illness, spinal muscular atrophy, medical healthcare concept
• Source: Shutterstock

It has been a bumpy road but Novartis has made a major leap in advancing an intrathecal version of its spinal muscular atrophy (SMA) gene therapy Zolgensma which would significantly expand the number of people who would be eligible to receive the transformational drug.

The Swiss major has presented positive topline results from a Phase III study which assessed OAV101 IT, the intrathecal formulation of Zolgensma (onasemnogene abeparvovec), in treatment-naïve patients with SMA type...

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