Efficacy of lenadogene nolparvovec gene therapy versus idebenone in Leber hereditary optic neuropathy due to the m.11778G>A MT-ND4 variant: two matching adjusted indirect comparisons.

No statistically significant difference was observed for time to initial clinically relevant recovery and change from baseline BCVA at 24 months.

For the matching adjusted indirect comparisons expanded access programme versus REFLECT, there was a low overlap between the two populations related to a difference in the time from vision loss to treatment.

In a post hoc sensitivity analysis comparing expanded access programme to RESCUE data (effective sample size: 33), clinically relevant recovery was 39.0% for idebenone versus 69.5% for lenadogene nolparvovec (OR=3.59, 95% CI 1.42 to 9.06; p=0.011).