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 £630,000 ($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. In clinical trials, injections of Luxturna restored eyesight to people with severe visual impairment due to retinal dystrophy. Spark estimates that up to 2,000 people in the US suffer from the condition, with the number of sufferers rising to 6,000 when Europe and other markets where it could sell the treatment are included.
Spark Therapeutics, the company behind the treatment, had previously claimed the treatment was worth $1m, citing the cost of a lifetime of blindness in lost earnings and wages for caregivers. But the firm said it had settled for the lower price of $850,000, or $425,000 per eye.
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. In clinical trials, injections of Luxturna restored eyesight to people with severe visual impairment due to retinal dystrophy. Spark estimates that up to 2,000 people in the US suffer from the condition, with the number of sufferers rising to 6,000 when Europe and other markets where it could sell the treatment are included.
Spark Therapeutics, the company behind the treatment, had previously claimed the treatment was worth $1m, citing the cost of a lifetime of blindness in lost earnings and wages for caregivers. But the firm said it had settled for the lower price of $850,000, or $425,000 per eye.
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.
Glybera, a gene therapy for a rare protein disorder, was launched in 2012 with a price tag of $1.2m. However, it was never approved in the US and was discontinued by manufacturers uniQure because of a lack of demand.
Soliris, a drug that treats a condition called paroxysmal nocturnal hemoglobinuria that attacks red blood cells, can cost up to $700,000 a year, while Elaprase, used in the treatment of Hunter syndrome, costs $500,000 a year.
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