GenSight Biologics reports validation of LUMEVOQ® Marketing Authorisation Application (MAA) by European Medicines Agency

GenSight Biologics reported that the LUMEVOQ® Marketing Authorisation Application (MAA) passed the validation checks required for submissions to the European Medicines Agency (EMA), triggering the official start of the MAA review procedure. The application for use of LUMEVOQ® gene therapy to treat vision loss in patients with Leber Hereditary Optic Neuropathy (LHON) due to a mutated ND4 mitochondrial gene was submitted in September, and the procedure was formally initiated on October 29.

Bernard Gilly, Co-founder and Chief Executive Officer of GenSight Commented “We are delighted that the formal review is now underway and look forward to clarifying any questions that the EMA’s scientific committees may have, Meanwhile, we continue apace with our commercial preparations to ensure that LUMEVOQ® becomes available to LHON patients at the earliest possible time.”

LHON is a rare, mitochondrial genetic disease, mainly affecting young males. The ND4 mutation results in the worst visual outcomes, with most patients becoming legally blind. There continues to be a high unmet medical need for the 800-1200 new ND4 LHON patients in Europe and the U.S. each year, particularly those who are struck blind in their prime working years.

Lenadogene nolparvovec (tradename: LUMEVOQ®) is a recombinant adeno-associated viral vector, serotype 2 (rAAV2/2), containing a cDNA encoding the human wild-type mitochondrial NADH dehydrogenase 4 protein (ND4), which was specifically developed for the treatment of LHON associated with mutation in the ND4 gene. GenSight submitted the MAA based on the benefit-risk balance established by results from a Phase I/IIa study (CLIN-01), two pivotal Phase III efficacy studies (CLIN-03A: RESCUE, and CLIN-03B: REVERSE) and the long-term follow up study of RESCUE and REVERSE (CLIN 06 – readout at Year 3 post injection), supported by a statistics-based indirect comparison methodology to establish the gene therapy’s efficacy compared to natural history.

The next major administrative milestone step in the review is a pause at Day 120, at which point the Committee for Advanced Therapies will issue a first round of questions and the Company responds as well as submitting information that had been pre-agreed with the agency.

The Company is also working towards submitting LUMEVOQ®’s Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) in H2 2021.

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