Give your opinion on two clinical trials with a genetically modified medicinal product for the treatment of spinal muscular atrophy

The FPS Public Health and the FAMHP invite you to participate in the public consultation on two clinical trials with the genetically modified medicinal product AVXS-101 from the company AveXis, Inc. The purpose of the trials is to evaluate the safety, tolerability and efficacy of AVXS-101 for the treatment of spinal muscular atrophy. The public consultation runs from 17 October 2018 to 17 November 2018 inclusive.

In the clinical trials, AVXS-101 is administered intravenously as a recombinant biological product that is comprised of a non-replicating, non-integrating recombinant self-complementary adeno-associated virus serotype 2/9 (AAV2/9) capsid shell containing the cDNA of the human survival motor neuron (SMN) gene. AVXS-101 encodes for the human SMN protein, the loss of which appears to be the root cause of spinal muscular atrophy. A previous clinical trial demonstrated a favourable safety profile for AVXS-101 with an improvement in motor function and nutritional status and with an achievement of developmental milestones. Thus, AveXis continues the development of AVXS-101 to improve disease course, prolong survival, and to delay the need for respiratory assistance in patients with spinal muscular atrophy.

This development program consists of two clinical trials.

  • In the first clinical trial, AVXS-101 is administered to babies of max 6 weeks (42 days) old with spinal muscular atrophy and with two to four copies of the SMN2 gene.
  • In the second clinical trial, AVXS-101 is administered to babies of max 6 months (180 days) old with spinal muscular atrophy type 1 and with one or two copies of the SMN2 gene.

The first clinical trial will be conducted at the Centre Hospitalier Régional Hôpital La Citadelle in Liège. The second clinical trial will be conducted at the University Hospital in Ghent and at the Centre Hospitalier Régional Hôpital La Citadelle in Liège.

As a citizen of Belgium you can comment on this clinical trial application.

For each clinical trial application related to the use of genetically modified organisms, the authority organises a public consultation for thirty days (in accordance with the Royal Decree of 21 February 2005).

You will have access to the data in the application file, as well as to an on-line form (see Dutch and French) to send comments or ask questions about this file.

The public consultation will last until 17 November 2018 inclusive.

General information on GMOs and public consultation (see Dutch and French)

 

Last updated on
17/10/2018