Issuance of a compassionate access authorization for GS010/LUMEVOQ®

GenSight Biologics has received compassionate access authorization for its gene therapy GS010/LUMEVOQ® from the French National Agency for Medicines and Health Products Safety (ANSM). This authorization allows patients with Leber Hereditary Optic Neuropathy (LHON) linked to the ND4 mutation to benefit from the treatment, despite the lack of marketing authorization. This provision applies to serious illnesses for which there are no therapeutic alternatives.

Compassionate access requires that the request be initiated by healthcare professionals, with prior assessment by the ANSM (French National Agency for Medicines and Health Products Safety). Eligibility criteria include the duration since the onset of vision loss. This advancement is intended for cases where the medical need is not currently being met and is based on a favorable benefit-risk ratio.