Diamond Pharma Services, a technical services and regulatory affairs consulting group, provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight Biologics, including the preparation, authoring support, agency communication and submission of GenSight’s first Marketing Authorization Application to the European Medicines Agency (EMA), for its novel ocular gene therapy LUMEVOQ. The EMA decision is expected in the second half of 2021.
 
LUMEVOQ (Lenadogene nolparvovec) is a gene therapy to treat vision loss due to the rare, mitochondrial genetic disease, Leber Hereditary Optic Neuropathy (LHON) caused by mutation in the ND4 mitochondrial gene. LHON mainly affects young males, and the ND4 mutation results in the worst visual outcomes, with most patients becoming legally blind. There is a high unmet medical need for LHON patients, of which there are 800-1200 in the EU and the U.S. annually.
 
Headquartered in Paris, France, GenSight is a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. Diamond has provided GenSight with regulatory, pharmacovigilance, quality and compliance support for LUMEVOQ leading up to the MAA assessment. 
 
“We are pleased GenSight Biologics chose to work with our team of experts at Diamond to provide European Regulatory, Pharmacovigilance, Quality and Compliance support for LUMEVOQ, its first MAA submission, and the first for a gene therapy treating a mitochondrial disease. It has been a personal ambition of mine to have the opportunity to work on a gene therapy within the ophthalmic arena, and GenSight has allowed Diamond that opportunity and that privilege,” said Maureen Graham, managing director, regulatory, Diamond Pharma Services. “This submission adds to Diamond’s broad experience in providing support to companies developing advanced therapy medicinal products (ATMPs), which includes over 50 programs at various stages of development, and two MAA approvals – Glybera and Yescarta.”