Cost of sight

A new gene therapy for the treatment of the inherited retinal dystrophy will come with a $850,000 (£627241) price tag.

Luxturna, developed by Spark Therapeutics, became the first gene therapy for an inherited disorder to receive approval from the US Food and Drug Administration in December.

Spark Therapeutics has reached an agreement in principle with Harvard Pilgrim and Express Scripts to manufacture the one-time treatment at a cost of $425,000 (£313,624) per eye.

The condition that Luxturna targets is retinal dystrophy due to a mutation of the RPE65 gene.

The safety and efficacy of Luxturna were established in a clinical development programme with 41 patients between the ages of four and 44 years.

The primary evidence of efficacy of Luxturna was based on a phase three study with 31 participants by measuring the change from baseline to one year in a subject’s ability to navigate an obstacle course at various light levels.

The group of patients that received Luxturna demonstrated significant improvements in their ability to complete the obstacle course at low light levels in comparison to the control group.