GenSight Biologics Granted Compassionate Access Authorization in France for its Gene Therapy
The French National Agency for the Safety of Medicines and Health Products has granted a compassionate access for GS010/LUMEVOQ, GenSight Biologics' gene therapy aimed at treating Leber's hereditary optic neuropathy, according to the company's statement.
Understanding Compassionate Access Authorization
Compassionate access authorization is a national mechanism that allows patients suffering from serious, rare, or disabling diseases to access treatments not yet authorized for marketing, the company explains. This procedure is applicable when there is an unmet medical need and no appropriate therapeutic alternative is available. To be eligible for compassionate access, the candidate treatment must demonstrate a favorable benefit-risk ratio, the statement specifies. Requests can only be initiated by healthcare professionals, who submit individual applications to the ANSM for identified patients. The Agency then evaluates these requests and decides on their authorization. According to GenSight Biologics, patients for whom a treatment request with GS010 is filed must meet specific eligibility criteria, particularly regarding the time elapsed since the onset of their vision loss.
About Leber's Hereditary Optic Neuropathy
Leber's hereditary optic neuropathy is a rare mitochondrial disease, maternally transmitted, characterized by the degeneration of retinal ganglion cells, the group states. It causes sudden and generally irreversible vision loss, often progressing to legal blindness, the statement indicates. Among the mutations involved, the mitochondrial ND4 mutation is the most common and is associated with the worst prognosis among the major mutations, the company specifies. GS010 LUMEVOQ, whose active principle name is lenadogene nolparvovec, relies on a proprietary mitochondrial targeting sequence technology derived from the work of the Vision Institute in Paris. The product has not received marketing authorization in any country and is not commercially available, GenSight Biologics reminds.
GenSight Biologics: A Clinical-Stage Biopharmaceutical Company
GenSight Biologics is a clinical-stage biopharmaceutical company specialized in the development and commercialization of gene therapies for neurodegenerative retinal diseases and central nervous system disorders, according to the statement. The company's portfolio is based on two key technological platforms, the Mitochondrial Targeting Sequence and optogenetics, the company indicates. Through its gene therapy-based approach, GenSight Biologics' candidates are designed to be administered via a single intravitreal injection per eye, the group specifies. The company is listed on Euronext Paris under the ticker SIGHT and is eligible for the PEA PME scheme.
