French biopharma company Gensight Biologics has submitted a marketing authorisation application for its lead gene therapy Lumevoq to the European Medicines Agency (EMA).
Gensight is aiming to win approval for Lumevoq (lenadogene nolparvovec) as a treatment for patients with vision loss due to Leber Hereditary Optic Neuropathy (LHON), caused by mutation in the ND4 mitochondrial gene.
Lumevoq is a recombinant adeno-associated viral vector which has been specifically developed for the treatment of LHON associated with a mutation found in the ND4 gene.
LHON is a rare genetic disease that overwhelmingly affects young males, with the ND4 mutation causing the worst visual outcomes for patients.
“This first regulatory submission for GenSight is a major milestone in our progression from a pure research organisation to one with commercial capabilities,” said Bernard Gilly, Co-founder and Chief Executive Officer of GenSight Biologics.
“It validates a technology platform that has the potential to address the high unmet medical needs of patients suffering from a range of rare diseases. I would like to thank all GenSight employees and partners whose motivation, focus and effort made this submission possible,” he added.
The EMA marketing application is based on results from a Phase I/IIa study and two Phase III efficacy studies, as well as long-term studies.
In the CLIN06 study, after two years post-treatment, patients experienced an average gain of +18.8 letters equivalent relative to the low point of their visual acuity in their Lumevoq treated eyes, compared to +17.3 letters equivalent in their sham treated eyes.
Three years after the one-time injection, the bilateral benefit was maintained with Lumevoq-treated eyes recording a mean improvement against patients’ equivalent relative to the low point of their visual acuity of +20.5 letters.