A 31-year-old Irishman has been able to read for the first time in more than a decade after becoming the country’s first patient to receive Luxturna (voretigene neparvovec) to treat RPE65-inherited retinal dystrophy.
For the 13 years prior to receiving treatment in November 2024 at Dublin’s Mater University Hospital, Stuart Haxell was only able to perceive a small amount of light. Within two weeks of the procedure he said he experienced significant improvement in his vision. “The doctor who was examining me held up a large letter close to my eyes and I was able to make it out clearly. I can now read using magnification software – something I absolutely would not have been able to do before the operation.”
Professor David Keegan, ophthalmic surgeon at Mater University Hospital, said inherited retinal dystrophies are very rare but his team has identified between five and 10 patients in the country with RPE65-inherited retinal dystrophy. With the treatment system now set up, a second patient is expected to receive Luxturna later this year, said Prof Keegan.
Funding for the treatment was provided by Ireland’s Health Service Executive in 2024. In New Zealand, Pharmac is reportedly re-considering Luxturna’s long-term efficacy data with regard to funding the treatment. “The Ophthalmology Advisory Committee recommended that voretigene neparvovec be listed with high priority for the treatment of RPE65-inherited retinal dystrophy within the context of eye diseases, subject to Special Authority criteria,” reported Pharmac via its application tracker in December.
