FDA approves Luxturna to treat patients with inherited retinal disease

The US Food and Drug Administration (FDA) has this week approved the first ever gene therapy treatment, Luxturna (Voretigene Neparvovec), for inherited retinal diseases (IRD).The gene therapy drug will be used as a treatment for patients with vision loss caused by RPE65 gene mutations.

Mutations in the RPE65 gene are associated with two main types of IRDs – retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA), where the mutation accounts for 2% and 6-16% of diagnoses respectively. Until now, there were no treatments for these two conditions.

Michele Acton, CEO of Fight for Sight, said: “Currently there are no treatments for these conditions and being able to provide patients with treatment options for the first time is very exciting. We need to ensure that novel treatments are made available to patients in the UK too. This is another development that demonstrates the necessity of funding innovative research.”

Scott Gottlieb, FDA Commissioner, said: "Today's approval marks another first in the field of gene therapy — both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss — and this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases."

The gene therapy has been developed by Spark Therapeutics. “Today’s landmark approval of LUXTURNA is a moment decades in the making for the field of gene therapy, the inherited retinal disease community, and most importantly, patients with biallelic RPE65 mutation associated retinal dystrophy who now have the option to seek treatment,” said Jeffrey D. Marrazzo, CEO at Spark Therapeutics.

In the UK, Luxturna has been subject to a NICE “draft scope” but has not so far been referred by the Department of Health to NICE for full appraisal.

Further information relating to this story can be found in our news item from August 2017: Data published from the first Phase 3 gene therapy clinical trial for an inherited retinal disease