AVXS-101 Receives “Breakthrough Therapy Designation”

28 July 2016

The US Food and Drug Administration (FDA) has granted “Breakthrough Therapy” status to AVXS-101, a gene therapy drug with future potential to treat SMA.

Produced by AveXis, AVXS-101 makes use of genetically engineered, harmless viruses to increase levels of the SMN protein, which is lacking in SMA patients.

The Breakthrough Therapy Designation has been granted based on early results from the ongoing Phase I, open-label clinical trial of AVXS-101. The latest details from the trial were released in May 2016, and indicate that AVXS-101 is generally well tolerated, safe, and causing positive improvements in the young SMA patients enrolled in the trial (click here for more information).

To be granted Breakthrough Therapy status a drug must be designed to treat a serious disease, and show an early indication in clinical trials of being able to provide substantial benefit to patients, both of which apply to AVXS-101.

This special designation from the FDA was devised to improve the communication between the FDA and the company developing the drug of interest. It means that the FDA will provide AveXis with advice on how to best proceed with the development of AVXS-101 in the hope that this will speed up the drug approval process.

AveXis has been requested by the FDA to submit what is called a Type B meeting request, which will facilitate interactions between the two organisations. AveXis are hoping to submit this request by the end of July 2016.


Further Information

AveXis press release

Accelerating the approval process

Breakthrough Therapy FAQ

AveXis clinical trial website