The US Food and Drug Administration (FDA) has fast-tracked and approved a novel gene therapy, Luxturna (voretigene neparvovec-rzyl), to treat patients with a rare form of inherited vision loss that can result in blindness.
The news was welcomed by advocacy group Retina International and international retina specialists, including Auckland-based Associate Professor Andrea Vincent.
“This represents a very exciting milestone in the treatment of inherited retinal diseases,” said A/Prof Vincent. “There is a complex surgical procedure that has been developed to deliver this gene therapy. Therefore, it is likely at this point, that any patient would have to travel overseas to receive this treatment, although expertise exists in Australia.”
Luxturna has been approved in the US for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy that leads to vision loss and may cause complete blindness. The condition is rare, with approximately 1,000 to 2,000 patients believed to suffer from it in the US and just a few in New Zealand. Only two individuals have been identified in the New Zealand Database of Inherited Retinal Diseases, confirmed A/Prof Vincent.
Though the new treatment was welcomed, many international commentators however, are questioning the cost of treatment. Manufacturer Spark Therapeutics announced the cost at a whopping US$425,000 per eye at the end of last year, not including surgery costs, reigniting the cost of medicines debate in the US.
