Paris-based GenSight Biologics announced it has reached an agreement with France’s National Agency for Medicines and Health Products Safety (ANSM) to open an early access program (AAC) for its gene therapy LUMEVOQ.
According to the company, the program enables eligible patients suffering from Leber Hereditary Optic Neuropathy (LHON) with an ND4 mutation to access LUMEVOQ ahead of formal marketing authorisation. LHON is a rare mitochondrial genetic disease that can lead to rapid and severe vision loss, primarily affecting young adults.
The AAC program replaces the previously authorised temporary authorization for use (ATU) program, which was suspended in 2021 after GenSight reported production issues. The company said that the ANSM’s approval reflects positive clinical data and recent manufacturing improvements.
LUMEVOQ remains under review by the European Medicines Agency (EMA) for potential conditional marketing authorisation. The company claims it continues to work with regulatory authorities in other countries to expand patient access.
GenSight said that the new AAC is expected to provide treatment to eligible LHON patients in France while the EMA review process is ongoing. The company also noted it has taken steps to strengthen its manufacturing and supply chain capabilities to meet expected demand.
No financial terms or estimates on patient numbers were disclosed by the company in its announcement.
LUMEVOQ is designed as a single intravitreal injection targeting the underlying mitochondrial DNA mutation in LHON patients.
