7 July 2021 - - The French Competent Authority, the National Agency for Medicines and Health Products Safety (Agence Nationale de Sécurité du Médicament et des produits de santé or ANSM), has granted a Cohort Temporary Authorization for Use for Lumevoq in the treatment of Leber Hereditary Optic Neuropathy caused by a mutated ND4 gene, French biopharmaceutical company GenSight Biologics (PAR: SIGHT) (Euronext: SIGHT) (ISIN: FR0013183985) (PEA-PME eligible) said.

Lumevoq was first approved for early access in France in December 2019 when the ANSM authorized a Named Patient ATU ("ATU Nominative" or ATUn) for the CHNO des Quinze-Vingts Hospital in Paris.

To date, 18 patients have been treated under an ATUn. Under Named Patient ATUs, physicians have to submit individual requests to the ANSM for each patient.

The Cohort ATU greatly simplifies the process by which patients gain access to Lumevoq prior to EU marketing authorization expected in H1 2022.

French hospital-based physicians, including those practicing outside the Quinze-Vingts Hospital in Paris, will now be able to request treatment for eligible patients directly from GenSight Biologics.

The ATUc also allows the company to monitor patients more systematically and to collect data that would allow the safety and efficacy of Lumevoq to be assessed for these patients.

Under the ATUc, GenSight Biologics will provide Lumevoq to hospitals at a price similar to that in the current ATUn.

LHON is a rare, maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in precipitous and irreversible vision loss typically leading to legal blindness.

The disease mainly affects adolescents and young adults, striking patients suddenly and painlessly. LHON causes the blindness of an estimated 1,200 to 1,500 new patients each year in the United States and Europe.

The ND4 mitochondrial mutation, which LUMEVOQ targets, is associated with the most severe clinical form of LHON, with poor overall visual outcomes.

Under the Cohort ATU, Lumevoq will be administered as a bilateral intravitreal injection to patients with vision loss due to LHON caused by a confirmed G11778A mutation in the ND4 mitochondrial gene.

In France, pharmaceutical products not yet granted Marketing Authorization and not recruiting for a clinical trial can only be used if the ANSM authorizes an ATU.

ATUs are reserved for products whose efficacy and safety are "strongly presumed" based on clinical trial data and whose therapeutic indication targets a serious, rare or disabling disease lacking appropriate treatment.

The products are innovative, and the company filing the application must commit to submitting a Marketing Authorisation Application within one year from the approval of the Cohort ATU.


A Cohort ATU is granted only after a company submits a successful application to the ANSM, detailing how patients will be treated and monitored in a Protocol for Therapeutic Use.

The authorization takes effect after the agency completes its review of the PUT and annexes submitted in the application. The granting of a Cohort ATU usually precedes the Marketing Authorisation.

GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders.

GenSight Biologics' pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases.

GenSight Biologics' lead product candidate, Lumevoq (GS010; lenadogene nolparvovec), has been submitted for marketing approval in Europe for the treatment of Leber Hereditary Optic Neuropathy, a rare mitochondrial disease affecting primarily teens and young adults that leads to irreversible blindness.