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GenSight Biologics has submitted a marketing authorization application to the European Medicines Agency for Lumevoq.
GenSight Biologics, a biopharma company focused on gene therapies for retinal neurodegenerative diseases and central nervous system disorders, has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for Lumevoq.
According to a Sept. 15, 2020 press release, the company has submitted the MAA, seeking approval for its lead product to be authorized for the treatment of patients with vision loss due to Leber Hereditary Optic Neuropathy (LHON) caused by mutation in the ND4 mitochondrial gene. The MAA has been submitted based on the benefit–risk ratio that was determined through results of a Phase I/IIa study, two Phase III studies, and long-term follow up studies. The company expects to submit a biologics license application for Lumevoq to the United States Food and Drug Administration in 2021.
Lumevoq (lenadogene nolparvovec) is a recombinant adeno-associated viral vector, serotype, which has been specifically developed for the treatment of LHON associated with mutation in the ND4 gene. The product was granted orphan drug designation by EMA in 2011 and by FDA in 2013.
“This first regulatory submission for GenSight is a major milestone in our progression from a pure research organization to one with commercial capabilities,” said Bernard Gilly, co-founder and CEO of GenSight Biologics, in the press release. “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.”
Source: GenSight Biologics