A revolutionary new gene therapy treatment that can save the eyesight of children who would otherwise go blind, is to be available on the NHS.

About 250 adults and 150 babies born with inherited retinal dystrophies disorder are registered in the UK each year. They have poor sight from birth which swiftly deteriorates, with most losing their vision completely in childhood.

Now the life changing therapy voretigene neparvovec will be available in the UK from 2020 after NHS England negotiated an undisclosed reduced price with manufacturer Novartis. The treatment normally costs £613,410 per patient.

It is the first in a new generation of gene therapies that can be directly administered, in this case through an injection.

Speaking at the Health Innovation Expo conference in Manchester, Simon Stevens, chief executive of NHS England said: “For previous generations, curing blindness would literally have been seen as a ‘miracle’. Now modern medicine is making that a reality for our patients.

“Loss of vision can have devastating effects, particularly for children and young people, but this is truly life-changing treatment restores the sight of people with this rare and distressing condition.

“Once again the NHS is at the forefront of the genomic revolution with patients in England among the first to benefit from this revolutionary new form of treatment.

“This latest deal reinforces the benefits for companies willing to engage with us and be pragmatic with their pricing – which is good news for patients, tax payers and industry.”

It is expected that up to 100 patients living with a retinal dystrophy to have access to treatment for the first time.

The condition blocks messages for making proteins in the eye that are essential for normal vision. The drug works by recreating these processes like that in a healthy functioning eye.

Sue Sharp, deputy chief executive at the Royal Society for Blind Children, (RSBC) 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.”

Robert MacLaren, professor of Ophthalmology at the University of Oxford and Consultant Ophthalmologist at the Oxford Eye Hospital, said: “The progression of inherited retinal degeneration caused by RPE65 gene mutations leads to blindness, which has a profound effect on the lives of affected patients and their carers.

“As a clinician, I believe the true value of voretigene neparvovec is its potential to improve vision in children and adults, and enabling them to participate fully at school, work and in their private lives.”

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