Posted by Michael Wonder on 16 Sep 2020
GenSight Biologics submits EU marketing authorisation application for Lumevoq gene therapy to treat vision loss due to Leber hereditary optic neuropathy
15 September 2020 - First marketing authorisation application for a gene therapy treating a mitochondrial disease.
GenSight Biologics today announced that it has submitted the marketing authorisation application for its lead product Lumevoq to the EMA, seeking approval for the treatment of patients with vision loss due to Leber hereditary optic neuropathy caused by mutation in the ND4 mitochondrial gene.
GenSight submitted the application based on the benefit-risk balance established by results from a Phase 1/2a study (CLIN-01), two pivotal Phase 3 efficacy studies (CLIN-03A: RESCUE, and CLIN-03B: REVERSE) and the long-term follow-up study of RESCUE and REVERSE (CLIN 06 - readout at Year 3 post injection).
