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GenSight Biologics Halts Lumevoq's Regulatory Process in Europe

GenSight Biologics Halts Lumevoq's Regulatory Process in Europe

April 26, 2023

GenSight Biologics announced that it is withdrawing its application submitted to the European Medicines Agency (EMA) seeking approval for Lumevoq (lenadogene nolparvovec) in patients with Leber hereditary optic neuropathy (LHON).

The decision comes after the Committee for Advanced Therapies (CAT) said that the company's data is insufficient to support a positive opinion on the marketing authorization of the gene therapy by the EMA.

In light of this, GenSight stated that withdrawing the application will enable the company to discuss the best possible path forward for Lumevoq with the EMA in the upcoming weeks, with the aim of submitting a new application that addresses remaining objections as soon as possible, in Europe and other countries. The company is exploring various options, including generating new clinical data, which may result in significant 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 has confirmed that its partner in the United States is on track with the manufacturing validation campaign, and the product will be released for human use as planned by the end of the year.

The submission of Lumevoq to the European Medicines Agency (EMA) by GenSight's Marketing Authorization Application (MAA) was based on the benefit-risk balance established by the results of clinical trials or compassionate use of 252 patients. This data set is unprecedented for ND4-LHON and shows that the efficacy of Lumevoq compares favorably to both natural history and idebenone.