Pharma Focus Europe

GenSight Biologics Issues Update Regarding Scientific Advice from the European Medicines Agency for LUMEVOQ®

Thursday, September 28, 2023

GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible) has provided an update regarding its discussions with the European Medicines Agency (EMA) on the design of a new Phase III trial for LUMEVOQ®. LUMEVOQ® is the company's gene therapy for LHON (Leber Hereditary Optic Neuropathy) caused by a mutated ND4 mitochondrial gene.

The forthcoming study, known as RECOVER, is set to be a randomized controlled trial comprising two arms: a sham control arm, where a simulated procedure mimics an injection into each eye without any substance being injected, and a treatment arm, where participants will receive bilateral intravitreal injections of LUMEVOQ® (also referred to as GS010). The EMA has indicated that the proposed study design involving bilateral administration is acceptable for assessing the benefits of GS010 in patients requiring treatment for both eyes. Furthermore, the study design includes a provision for open-label treatment, allowing individuals in the sham arm to receive LUMEVOQ® bilateral injections if the primary endpoint is met.

In addition to providing feedback on statistical analyses, the EMA has offered guidance on various aspects of the study design that GenSight will consider as it finalizes the plan.

The RECOVER study is intended to address concerns raised by the EMA’s Committee for Advanced Therapies (CAT) during the review of the Marketing Authorization Application (MAA) submitted in 2020. GenSight decided to withdraw the application in April 2023 to enable a more comprehensive discussion of the Agency’s concerns. Recruitment for the RECOVER study can commence once the study design is finalized, the product is manufactured and approved for human use, and approvals are obtained from local competent authorities and ethics committees. GenSight anticipates starting the study in the second quarter of 2024, with completion expected by the second half of 2026.

Bernard Gilly, Chief Executive Officer and Co-Founder of GenSight Biologics, expressed gratitude for the endorsement of key aspects of RECOVER and reiterated the company's commitment to providing timely access to LHON patients while meeting regulatory standards.

GenSight also plans to share the core elements of the RECOVER study design with other regulatory bodies, including the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and the U.S. Food and Drug Administration (FDA). An initial discussion with the MHRA is scheduled for November 2023, and the company intends to engage with the FDA in the near future to ensure that the results of the RECOVER study gain acceptance from all major regulatory authorities.

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