AveXis has submitted marketing authorisation to the US, European and Japanese health authorities for its gene therapy product, AVXS-101, for spinal muscular atrophy (SMA) type 1. If the file is accepted by the different health authorities, the decision is expected for the beginning of 2019, for the United States and Japan) or the mid-2019 for Europe.
On December 3, 2018, Novartis, which recently bought AveXis and continues to develop AVXS-101 – now known as Zolgensma – has just announced that the US Food and Drug Administration (FDA) has validated the filing of the file and would carry out a priority review of the product
This request follows positive results from a Phase I trial of intravenous AVXS-101 in 15 infants with SMA type 1. Product administration was well tolerated and lead to an improvement in motor function and development.
Other ongoing trials of AVXS-101 in SMA will provide additional data for the future marketing of AVXS-101 in SMA type 1 and subsequently, in SMA type 2. One of the trials, STR1VE EU, is a phase III trial that is taking place in Europe. It will soon include participants from the I-Motion Institute, Trousseau Hospital, Paris.
As a reminder, it was in 2009 and 2011 that researchers at Genethon and the Institute of Myology demonstrated for the first time the effectiveness of gene therapy in mouse models of SMA. In March 2018, Genethon granted AveXis a license to use the patents related to AAV9-SMN and its in vivo administration in the central nervous system, either intrathecally or intravenously.
Read the article from Cure SMA, October 18, 2018: “AveXis Files for FDA Approval of Gene Therapy for Spinal Muscular Atrophy Type I“
