GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced the submission of the updated regulatory file for LUMEVOQ® gene therapy to the French medicines safety agency Agence Nationale de Sécurité du Médicament et des Produits de Santé (ANSM) to prepare for the restart of the early access (AAC) program in France.
The submission documents the successful manufacture of LUMEVOQ®, including the blending of two GMP drug substance batches to optimize the number of vials available for clinical use and the passing of all required quality control tests. LUMEVOQ® is being developed as a treatment for Leber Hereditary Optic Neuropathy (LHON) caused by a mutated ND4 mitochondrial gene, a rare mitochondrial genetic disease that causes acute and usually irreversible loss of vision.
“With the submission, we mark the successful transition from manufacturing to regulatory review,” commented Laurence Rodriguez, GenSight Biologics Chief Executive Officer. “We join the LHON community in looking forward to the expeditious resumption of early access to LUMEVOQ by year’s end.”
The updated regulatory file will support the ANSM’s assessment of individual applications for compassionate use, which can now be submitted by healthcare professionals under the rules of the AAC program. GenSight Biologics anticipates a review period for these applications and will work closely with the ANSM to optimize the assessment timeline.
GenSight Biologics is preparing to initiate the supply of the drug to the designated treatment center, the Quinze-Vingts hospital in Paris, in mid-December. In parallel, the Company is working with the hospital’s administrative and medical teams to enable the first injections to be administered by the end of December.
