NHS to offer £600k ‘miracle’ cure for infant blindness

A “MIRACLE” jab that cures a rare form of child blindness will be offered by the NHS, its chief executive is expected to announce today.

Previously, no treatment has been available for infants born with inherited retinal dystrophy disorders, which cause poor vision and can result in complete blindness by childhood. Simon Stevens, the chief executive of the NHS, will announce the revolutionary gene therapy treatment at the Health Innovation Expo conference in Manchester.

He will say: “This truly life-changing treatment restores the sight of people with this rare and distressing condition. For previous generations, curing blindness would have been seen as a ‘miracle’. Now modern medicine is making that a reality for our patients.”

Retinal dystrophy comprises a range of disorders, such as retinitis pigmentosa. The treatment will be provided to patients with a specific faulty gene, RPE65, which blocks the messages that make proteins in the eye that are essential for normal vision. It is estimated up to 100 patients will be eligible for the new treatment. Around three to five babies a year are born with the condition.

NHS England reached a deal with manufacturer Novartis to fund the drug, which costs £613,410 per patient, at full price. The treatment is administered by a single injection to the back of each eye under the retina. Access is expected to be in place from January next year.

Sue Sharp, deputy chief executive at the Royal Society for Blind Children, said: “We see first hand the devastating effect of childhood sight loss, and so we welcome news of this breakthrough therapy and its impact on the lives of children with inherited retinal dystrophies disorder.”