GenSight Biologics announced that it has submitted the Marketing Authorization Application (MAA) for its lead product Lumevoq to the European Medicines Agency (EMA), seeking approval for the treatment of patients with vision loss due to Leber Hereditary Optic Neuropathy (LHON) caused by mutation in the ND4 mitochondrial gene.
“This first regulatory submission for GenSight is a major milestone in our progression from a pure research organization to one with commercial capabilities. 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,” Bernard Gilly, Co-founder and Chief Executive Officer of GenSight Biologics, said in a company news release.
LHON is a rare, mitochondrial genetic disease, mainly affecting young males. The ND4 mutation results in the worst visual outcomes, with most patients becoming legally blind. There continues to be a high unmet medical need for the 800-1200 new LHON patients in Europe and the U.S. each year, particularly those who are struck blind in their prime working years.
Lenadogene nolparvovec (tradename: Lumevoq) is a recombinant adeno-associated viral vector, serotype 2 (rAAV2/2), containing a cDNA encoding the human wild-type mitochondrial NADH dehydrogenase 4 protein (ND4), which has been specifically developed for the treatment of LHON associated with mutation in the ND4 gene. It received orphan drug designation status for the treatment of LHON from the EMA in 2011 and from the FDA in 2013.
GenSight submitted the MAA 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). To demonstrate the efficacy of Lumevoq in the context of a contralateral effect, the company used a statistics-based indirect comparison methodology to assess the visual outcomes in Lumevoq-treated patients (from Lumevoq efficacy studies) against those in untreated patients from Natural History studies and GenSight’s REALITY Natural History Registry.
GenSight expects to submit the Biologics License Application (BLA) for Lumevoq to the FDA in H2 2021. First-in-human data from GenSight’s second clinical stage program, GS030, are expected to be available in H2 2021.