Research & Development
Novartis receives US FDA priority review for Zolgensma
5 December 2018 -

Switzerland-based Novartis has received a US Food and Drug Administration (FDA) priority review for its gene therapy Zolgensma (onasemnogene abeparvovec-xxxx), which was formerly known as AVXS-101, intended for the treatment of spinal muscular atrophy Type 1, it was reported on Tuesday.

Zolgensma was earlier granted breakthrough therapy designation by the US regulator. The product, which is intended to be delivered as a single, one-time infusion, functions by replacing the missing or defective SMN1 gene with a functional copy that makes SMN protein. Through this mechanism, the gene therapy is said to help in improving motor neuron function and survival.

The company expects a regulatory action to be taken on the gene replacement therapy in May 2019. The investigational gene therapy was originally developed by AveXis, a US-based clinical stage gene therapy company that Novartis purchased for USD8.7bn in an all-cash deal this year.



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