GenSight Biologics Withdraws its EMA Application for Lumevoq
GenSight Biologics announced that it has decided to withdraw an application with the European Medicines Agency (EMA) seeking approval of Lumevoq (lenadogene nolparvovec) in patients with Leber hereditary optic neuropathy (LHON). The moves comes after the Committee for Advanced Therapies (CAT) indicated that the company's data is not sufficient to support a positive opinion of the marketing authorization of the gene therapy by the EMA.
GenSight said this decision enables the the company to discuss the best possible path forward for Lumevoq with the EMA in the coming weeks, aiming at submitting a new application addressing remaining objections as soon as possible, in Europe and other countries. The company said it is exploring options including generating new clinical data, which may induce material delays and additional costs.
“GenSight teams have gathered a huge dataset of 252 ND4-LHON patients treated with Lumevoq showing that 70% of Lumevoq-treated patients present a visual recovery, in contrast to the poor and limited recovery observed in the natural history of the disease,” José-Alain Sahel, Co-founder of GenSight Biologics and of the Institut de la Vision, Paris, France, said in a company news release. “In light of the study results, confirmed by real-life data, Lumevoq is the current best therapeutic option for ND4-LHON patients given the 3-fold difference in vision function in treated patients. It is disappointing that the contralateral effect of the therapy limited the perceived strength of these data, published in top-tier peer-reviewed journals by leaders in the field.”
“We disagree with the current CAT assessment and remain highly confident in the clinical benefit of Lumevoq for LHON patients, which is supported by extensive evidence from multiple clinical trials and real-world data,” said Bernard Gilly, Chief Executive Officer and Co-Founder of GenSight Biologics. “The decision to withdraw our application allows us to continue to work with EMA to agree as soon as possible on a regulatory path forward. I want to thank the patient communities for their support and reaffirm our determination to bring this innovative therapy to ND4-LHON patients in need of an efficacious treatment. I also want to thank the scientific community and our teams for their long-lasting commitment.”
GenSight confirms that the manufacturing validation campaign is on track with its partner in the United-States, as planned, with a product released for human use by the end of the year.
GenSight's Marketing Authorization Application (MAA) submission of Lumevoq to the EMA was based on the benefit-risk balance established by results from 252 patients treated through clinical trials or compassionate use. This unprecedented data set for ND4-LHON establishes that Lumevoq efficacy compares positively to both natural history and idebenone.