Gene therapy for rare form of blindness wins US approval

PHOTO COURTESY OF WIKIMEDIA

By MATTHEW PERRONE
AP Health Writer

WASHINGTON (AP) – U.S. health officials have approved a breakthrough therapy that can improve the eyesight of patients with a rare form of inherited blindness.

Tuesday’s approval of the treatment from Spark Therapeutics is another major advance for the emerging field of genetic medicine. However, the drugmaker says it will not disclose the price until next year, delaying potential debate about a treatment that Wall Street analysts expect to cost around $1 million.

The Food and Drug Administration approved the drug, Luxturna (Lux-turn-a) for a rare vision-destroying mutation that affects up to 2,000 people in the U.S. Luxturna is the first gene therapy in the U.S. for an inherited disease.