Novartis announced today that the European Commission (EC) 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 US 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.”
“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,” said Dr Bart Leroy, ophthalmologist and clinical geneticist, Professor and Head, Department of Ophthalmology at Ghent University Hospital, Ghent, Belgium and Director of Retinal Degenerations Clinic at Children’s Hospital of Philadelphia, Philadelphia, PA. “After more than 20 years of gene therapy research, there is finally a promising future ahead for the treatment of rare genetic eye disorders.”