Cost of treating rare genetic blindness: $425,000 per eye

By Ed Adamczyk Contact the Author   |  Jan. 3, 2018 at 3:08 PM
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Jan. 3 (UPI) -- Treatment of a rare, inherited form of blindness will cost $425,000 per eye, or $850,000 per pair, the company bringing the therapy to market announced on Wednesday.

Spark Therapeutics, a small, Philadelphia-based biotechnology company, received approval from the U.S. Food and Drug Administration in December for Luxturna.

Luxturna treats biallelic RPE65 mutation-associated retinal dystrophy, a retinal disease which leads to blindness, and is among the first in a series of gene therapy medicines expected to provide results after a single treatment. Between 1,000 and 2,000 people in the United States are estimated to have the disease.

Gene therapy involves delivering a healthy copy of a gene to replace a deficient one, in this case the RPE65 gene. The mutation in the gene can lead to impaired vision, which often deteriotes to blindness.

After the FDA approval, speculation turned to its price. The treatment for one pair of eyes was expected by Wall Street to be around $1 million, Bloomberg Technology reported on Wednesday, with the final announced price coming in at $425,000 per eye -- just short of the prediction.

Pharmaceutical companies are typically funded by regular purchases of treatments and medications for chronic conditions. Payment for the new therapy will be arranged by tying payments to the therapy's effectiveness, and will include options such as installments.

The company said it is discussing payment over time with the U.S. Centers for Medicare & Medicaid Services, as well as with pharmaceutical fulfillment company Express Scripts Holding Company.

Spark already has a program in place with Boston insurer Harvard Pilgrim Health Care, by which Spark will get a full payment immediately after treatment and offer a rebate if patients do not see an immediate benefit to their eyesight, measured at 30 days or 30 months.

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