Novartis’ Luxturna, a one-time gene therapy for the treatment of patients with vision loss due to a genetic mutation, developed and commercialized in the US by Spark Therapeutics, has been approved by European Commission (EC) in EU, as well as Iceland, Liechtenstein and Norway.
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 blindness3. Research shows that vision impairment and blindness in children frequently cause social isolation, emotional distress, loss of independence, or hazards such as falls and injuries5. The working copy of the RPE65 gene provided by Luxturna can restore vision and improve sight in children and adults with sufficient viable retinal cells3.
“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.”
Dr Bart Leroy, ophthalmologist and clinical geneticist, Professor of Ophthalmology at Ghent University Hospital, in Belgium said: “As a clinician who has worked for over 20 years with patients with inherited retinal disease and their families, I’ve seen firsthand the profound impact blindness can have on quality of life. It’s exciting to practice medicine at a time when we can offer options to children and adults facing blindness. After more than 20 years of gene therapy research, there is finally a promising future ahead for the treatment of rare genetic eye disorders.”
Paul Hudson, CEO, Novartis Pharmaceuticals, said: “EU approval of the one-time gene therapy Luxturna marks a milestone in reimagining medicine and can bring real value to patients, their families and society as a whole. Novartis is committed to working with patients, caregivers, health systems and physicians to establish access to this gene therapy for RPE65 patients, as we believe it can help restore sight and improve vision in children and adults who currently have no treatment options.”