The European Commission (EC) has approved Luxturna, a one-time gene therapy for the treatment of patients with vision loss due to a genetic mutation in both copies of the RPE65 gene and who have enough viable retinal cells.
The authorization is valid in all 28 member states of the EU, as well as Iceland, Liechtenstein, and Norway. Luxturna was developed and is commercialized in the U.S. by Spark Therapeutics.
People born with mutations in both copies of the RPE65 gene can experience profound sight loss from an early age, with the majority of patients progressing to total blindness. Research shows that vision impairment and blindness in children frequently cause social isolation, emotional distress, loss of independence, or hazards such as falls and injuries. The working copy of the RPE65 gene provided by Luxturna can restore vision and improve sight in children and adults with sufficient viable retinal cells.
“Today’s approval is momentous for patients given that there have been no pharmacological treatment options to date to treat this form of LCA,” said Christina Fasser, president of Retina International, an umbrella organization of more than 43 patient organizations worldwide promoting research to find treatments for inherited retinal degenerative diseases. “Access to this treatment has the potential to reduce the substantial physical, emotional and financial burden this disease has on patients and their families.”
The EC decision is based on a positive CHMP opinion that looked at data from a Phase 1 clinical trial, its follow-up trial, and the first randomized, controlled Phase 3 gene therapy trial for an inherited disease. In the Phase 3 clinical trial vision improvement was recorded as early as 30 days following treatment.
At 1 year, compared to the control group, patients treated with voretigene neparvovec improved by 1.6 light levels on the binocular multi-luminance mobility test (MLMT), the trial’s novel, patient-centric, primary endpoint. Vision improved by one or more light levels for 90 percent of patients treated with voretigene neparvovec, and 65 percent were able to successfully navigate the MLMT at the lowest light level of 1 lux at 1 year.
Decisions from national reimbursement bodies on Luxturna for patients with vision loss due to a genetic mutation in both copies of the RPE65 gene are expected in 2019 and 2020.
Novartis announced it is working closely with stakeholders to help ensure that eligible patients can start benefiting from this treatment as quickly as possible once reimbursement decisions are available in 2019 and 2020. A range of resources and reimbursement and access approaches are also being explored in order to provide support to both patients and healthcare professionals.