A new treatment for a rare form of blindness received approval from the Food and Drug Administration in December, and the pharmaceutical company behind it, Spark Therapeutics, has set the price of the one-time treatment at $450,000 an eye. Previously it had been reported that the makers of Luxturna, a gene therapy drug that is said to cure an inherited retinal disease, believed the treatment was worth $1 million, but after many health insurers voiced concern about affordability, the company has decided on a more discounted rate than what it had originally planned.
The drug will be available in the United States in early spring and is intended for patients with retinal dystrophy due to a mutation of the RPE65 gene. Currently there are about 1,000 to 2,000 people in the United States who could benefit from Luxturna, a gene therapy that supplies a normal RPE65 gene into the cells of the retina. The treatment does not promise 20/20 vision, but the drug makers say it returns sight to those with this rare form of blindness.
During the clinical trials, 27 out of 29 patients who received the gene therapy regained functional vision. Luxturna is a liquid treatment that is injected directly into the eyes and because the gene mutation typically affects both eyes, most patients would need treatment for both. Spark Therapeutic says it is working on offering payment plans to insurers and have also considered offering pricing that is dependent on how well the drug works to return sight for each individual patient. A press release from the company states, “Based on feedback from payers, Spark Therapeutics has been seeking solutions that would allow customers to pay for Luxturna in installments over several years rather than in a single, up-front payment.”
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