Luxturna

Luxturna is a medicine that is used to treat adults and children with loss of vision due to inherited retinal dystrophy, a rare genetic disorder of the retina (the light sensitive membrane at the back of the eye). Luxturna can only be used while patients still have enough functioning cells left in the retina and when the disease is caused by mutations (changes) in the gene RPE65. RPE65 is responsible for the production of an enzyme called all-trans retinyl isomerase, which is necessary for the normal functioning of retinal cells. Luxturna contains the active substance voretigene neparvovec and is a type of advanced therapy medicine called a ‘gene therapy product’. This is a type of medicine that works by delivering genes into the body. Inherited retinal dystrophy is rare, and Luxturna was designated an ‘orphan medicine’ (a medicine used in rare diseases) for two forms of the disease on various dates (retinitis pigmentosa: 28 July 2015; Leber's congenital amaurosis: 2 April 2012).