US drug firm says it can cure blindness - but it’s going to cost an arm and a leg
Spark Therapeutics says US$425,000 per eye is a reasonable price to pay for its gene-therapy injections
A drug whose inventors claim it can cure a rare form of blindness is to be one of the most expensive medicines ever sold at US$850,000.
Luxturna is injected directly into the eye to address the root cause of visual impairment by replacing a defective gene in the retina. It is the first gene therapy to be approved for use in the US, and was given the go-ahead by the Federal Drug Administration last month. However, the price of the treatment has only just been revealed.
“We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients,” said Spark Therapeutics’ chief executive, Jeff Marrazzo.
Luxturna is one of an emerging breed of gene therapies that differ from more established medicines administered over a period of time. Such treatments involve a one-off procedure to alter defective DNA, allowing the body to repair the problem itself.
They include a treatment for haemophilia and another for so-called “bubble baby” syndrome, where sufferers have to live in a sterile environment, which is to be offered on the NHS despite a £500,000 price tag.