NEW YORK – GenSight Biologics has withdrawn its application to the European Medicines Agency for Lumevoq (lenadogene nolparvovec), a gene therapy for a rare inherited mitochondrial disorder associated with vision loss, the biopharmaceutical company said Thursday.
GenSight is developing Lumevoq as a treatment for Leber hereditary optic neuropathy (LHON), using an adeno-associated virus (AAV) vector that delivers a normal ND4 gene, mutations in which are known to cause the disorder.
French gene therapy developer GenSight withdrew its regulatory application for the drug on the heels of an assessment by the EMA's Committee for Advanced Therapies (CAT), which had indicated that data the firm had provided so far wouldn't be sufficient to support market authorization for Lumevoq. GenSight said it withdrew its application before the EMA's Committee for Medicinal Products for Human Use issued its final opinion based on the CAT assessment.
"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," GenSight CEO and Cofounder Bernard Gilly said in a statement. "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."
Although Phase III trial results published in 2021 demonstrated an improvement in visual acuity from baseline for Lumevoq-treated patients, the study failed to meet its primary endpoint after comparing the visual acuity change from baseline between participants who received Lumevoq in both eyes, versus those who received Lumevoq in one eye and placebo in another. Participants treated with Lumevoq in only one eye experienced a contralateral effect in their other eye, which reduced outcome differences between the two study groups, the company reported at the time.
"It is disappointing that the contralateral effect of the therapy limited the perceived strength of these data," GenSight Cofounder José-Alain Sahel said in a statement.
GenSight said it plans to discuss next steps with the EMA in the coming weeks, which could include generating new clinical data, and to submit another application.