Page last updated: 22nd June 2017

AveXis has announced that the process to manufacture their gene therapy drug for SMA, AVXS-101, is now aligned with expectations of the US Food and Drug Administration (FDA).

Unlike Nusinersen and other drugs with potential to treat SMA, AVXS-101 uses harmless, genetically engineered viruses to increase SMN protein levels. The process of making these viruses requires specific techniques and processes that do not necessarily apply to the production of other drugs in the SMA pipeline.

As viruses are infectious agents and inherently complex, their production for use as a gene therapy in humans requires strict regulations. Good Manufacturing Practice (GMP) is a system to ensure products like AVXS-101 are consistently produced according to high quality standards, thereby reducing the inherent risks associated with their administration.

Last year, AveXis submitted a Type B meeting request to the FDA, with the aim of facilitating interactions between the two organisations. This meeting was recently held and resulted in an agreement on the GMP commercial manufacturing process for AVXS-101.

The FDA will provide support to AveXis for the proposed manufacturing process, analysis methods, and validation of AVXS-101. Furthermore, guidance will be given on how best to assess how similar the GMP-derived product is to the original virus used in the START Phase 1 AVXS-101 trial in SMA patients.

New pivotal trials in SMA Type 1 patients and a Phase 1/2a trial in SMA Type 2 patients were recently announced for AVXS-101. In order for these trials to commence, AveXis is required to address a number of the FDA’s concerns regarding the GMP process. AveXis have stated that they are on track to complete the necessary amendments within the August timeframe.

AveXis have a fully-operational manufacturing facility dedicated to the production of GMP-compliant AVXS-101, which will produce viruses for the upcoming clinical trials pending FDA review.

The formulation of the GMP for AVXS-101 production will help to ensure that patients receive a safe and effective product.