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 AAV5-hFVIII-SQ (BMN 270) against hemophilia A. The public consultation runs from 27 February 2019 to 29 March 2019 inclusive.

Hemophilia A (HA) is a bleeding disorder caused by deficiency in the coagulation factor VIII (FVIII). The clinical manifestations of severe HA are frequent spontaneous bleeding episodes, with a substantially increased risk of death when the brain is involved. The purpose of the trials is to assess whether, in an expanded sample, AAV5-hFVIII-SQ can safely alter the clinical phenotype of hemophilia A patients.

The medicinal product AAV5-hFVIII-SQ, developed by BioMarin Pharmaceutical Inc., will be delivered by single intravenous dose. The clinical trials will be conducted at Universitair Ziekenhuis Antwerpen, Universitair Ziekenhuis Leuven and Cliniques Universitaires Saint-Luc in Brussels.

As a citizen of Belgium you can comment on this clinical trials application. For each clinical trial application related to the use of genetically modified organisms, the authority organises a public consultation for 30 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 (HTML) (in Dutch and French) to send comments or ask questions about this file.

The public consultation will last until 29 March 2019 inclusive.

General information on GMOs and public consultation (HTML) (in Dutch and French).