Okaz/Saudi Gazette

RIYADH – The Saudi Food and Drug Authority (SFDA) has approved registration of a genetic medicine for the treatment of hereditary loss of sight that could lead to blindness.

This cutting-edge genetic treatment is the Luxturna drug “voretigene neparvovec”.

Luxturna, the revolutionary new form of treatment, is deemed the first genetic treatment in Saudi Arabia that will save people with a rare inherited disorder from going blind. The disorder is known as inherited retinal disorder.

Luxturna has been approved for the treatment of adults and children suffering from sight loss due to inherited retinal disorder caused by genetic mutation of the gene “RPE65”. The genetic mutation prevents the body from producing a protein that is necessary for vision (sight), hence, leading to sight loss and ultimately to entire loss of vision and blindness.

The SFDA said that the effective substance in the drug Luxturna (voretigene neparvovec) is a modified virus containing an active replica of the gene “RPE65”. After injecting the substance, this gene is transferred to the retina cells (the layer in the rear part of the eye that can identify light). This enables the retina to produce the protein necessary for vision. The virus used for transferring the gene does not cause any disease to humans.

Luxturna is administered under anesthesia by surgeons specialized in retina surgery. The patient will take one shot in each eye on two separate days, with a gap of at least six days between the two operations.

Luxturna is injected directly under the retina. The SFDA has confirmed that tests have been conducted on the efficacy and safety of Luxturna, based on a third degree clinical study conducted on 31 volunteers. It was noticed that the vision of the patients, who used Luxturna, improved as seen from their ability to move from one place to another and follow a specific route fitted with dim lights, the SFDA said.