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AVXS-101 to receive expedited regulatory review in the US and Europe

06 December 2018

Novartis has announced that the US Food and Drug Administration (FDA) has accepted for review with Priority Status their new drug application for the experimental gene therapy for SMA, AVXS-101, which has now been renamed ZOLGENSMA.

This means that the FDA will very likely make a regulatory decision on the suitability of ZOLGENSMA for the treatment of SMA in the US in May next year.

This news comes in response to the Biologics Licence Application (BLA) submitted by Novartis to the FDA in mid-October this year (click here for more information)

ZOLGENSMA, which has Breakthrough Therapy status to facilitate the drug approval process in the US (for more information click here), uses harmless, genetically engineered viruses to increase SMN protein levels.

A Phase I trial of ZOLGENSMA with SMA Type 1 patients was completed in mid 2017 and showed that injection of the therapy into the bloodstream is safe, well tolerated, and able to improve and help sustain muscle function in young infants with the condition (for more information click here). Additional trials are now underway (click here for more information).

ZOLGENSMA also has PRIME (PRIority MEdicines) designation in the European Union, similar to the FDA Breakthrough Therapy status.

Similar to the BLA in the US, Novartis have submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for ZOLGENSMA. A regulatory decision from the EMA is anticipated in mid 2019.
 

Further Information

AveXis Press Release

Novartis