GenSight Biologics to withdraw application to EMA for Lumevoq as data is insufficient to support a CHMP opinion for LHON.

As provided by the review procedure and following responses to the D180 questions, an oral explanation was held on April 19. GenSight invited the world renowned LHON experts, Patrick Yu-Wai-Man, MD, PhD (Cambridge University, UK) and José-Alain Sahel, MD (University of Pittsburgh School of Medicine, USA) to share their clinical practice and perspective on Lumevoq data.

Following interactions with the CAT indicating that the data provided thus far would not be sufficient to support a positive opinion of the marketing authorization of Lumevoq by EMA, GenSight decided to withdraw its application ahead of a final opinion by the CAT. This decision enables 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 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,” said José-Alain Sahel, Co-founder of GenSight Biologics and of the Institut de la Vision, Paris, France, “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,” commented 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.”