Industry News
Luxturna Approved To Treat Inherited Retinal Dystrophy
The Australian Therapeutic Goods Administration (TGA) has approved Novartis' Luxturna for the following therapeutic use:
- Luxturna is indicated for the treatment of patients with inherited retinal dystrophy caused by pathological biallelic RPE65 mutations and who have sufficient viable retinal cells as determined by the treating physician.
- Pathological mutations of RPE65 should be confirmed by a National Association of Testing Authorities (NATA) or International Laboratory Accreditation Cooperation (ILAC) accredited laboratory.
Luxturna is designed to deliver a normal copy of the gene encoding the human retinal pigment epithelial 65 kDa protein (RPE65) to cells of the retina in persons with reduced or absent levels of biologically active RPE65. The RPE65 is produced in the retinal pigment epithelial (RPE) cells and converts all-trans-retinol to 11-cis-retinol, which subsequently forms the chromophore, 11 cis-retinal, during the visual (retinoid) cycle. The visual cycle is critical in phototransduction, which refers to the biological conversion of a photon of light into an electrical signal in the retina. Mutations in the RPE65 gene lead to reduced or absent levels of RPE65 isomerohydrolase activity, blocking the visual cycle, resulting in impairment of vision and ultimately complete blindness.
