GenSight Biologics has obtained the Cohort Temporary Authorisation (ATUc) from the French regulatory agency for its Lumevoq to treat a type of Leber Hereditary Optic Neuropathy (LHON).
The French National Agency for Medicines and Health Products Safety (ANSM) has indicated Lumevoq to treat patients with LHON, caused by a mutated ND4 gene.
Lumevoq (lenadogene nolparvovec) works by leveraging a mitochondrial targeting sequence (MTS) technology platform, developed at the Institut de la Vision in Paris.
Under the ATUc, the drug will be administered as a bilateral intravitreal injection to patients with vision loss due to LHON caused by a mutation in the ND4 mitochondrial gene.
GenSight co-founder and CEO Bernard Gilly said: “The decision of the French ANSM to authorize Lumevoq to be administered under a Cohort ATU will facilitate early access to treatment for patients with LHON and indeed attests to the safety and efficacy of Lumevoq.
“The compassionate use and expanded access programs already running in Europe and the US will also allow GenSight to collect additional data that will bolster the already impressive evidence on Lumevoq’s clinical benefit and the safety profile and support our drive to obtain marketing authorisation in Europe and North America.
“GenSight’s achievement of the Cohort ATU highlights the importance of several initiatives contained in the Health Innovation 2030 plan that was recently announced by the French President to support the growth of innovative healthcare startups.”
LHON is a rare mitochondrial genetic disease that involves damage of retinal ganglion cells and results in precipitous and irreversible vision loss which may lead to legal blindness.
Lumevoq targets the ND4 mitochondrial mutation, which is linked to the most severe clinical form of LHON, with poor overall visual outcomes.
The drug was initially approved for early access in France, with a Named Patient ATU from the ANSM, for the CHNO des Quinze-Vingts Hospital in Paris, in December 2019.
Named Patient ATUs require physicians to submit individual requests to the ANSM for the treatment of each patient, while Cohort ATU would simplify the access Lumevoq.
The ATUc allows French hospital-based physicians, including those outside the Quinze-Vingts Hospital in Paris, to request treatment directly from GenSight Biologics.
Also, it allows the company to monitor patients more systematically, collect data that supports the safety and efficacy of Lumevoq, to be assessed in the population.
