6 September 2021 - GenSight Biologics today announced that its gene therapy Lumevoq has been granted Promising Innovative Medicine designation by the UK’s MHRA for the treatment of vision loss due to Leber hereditary optic neuropathy caused by a confirmed G11778A mutation in the ND4 mitochondrial gene.

Promising Innovative Medicine designation is given to a medicinal product that is likely to offer a major advantage for patients. Promising Innovative Medicine designation is also an early indication that a medicine is a promising candidate for the MHRA’s Early Access to Medicines Scheme in the treatment, diagnosis or prevention of life-threatening or seriously debilitating conditions with an unmet need. 

The Early Access to Medicines Scheme is similar to the Cohort ATU (Autorisation Temporaire d’Utilisation de Cohorte [Cohort Temporary Authorisation for use]) program in France, which simplifies the process by which patients could be treated with promising treatments ahead of market authorisation. Lumevoq was approved for a French Cohort ATU in July 2021.

Read GenSight Biologics press release