Gene therapy for rare form of blindness approved

WASHINGTON — U.S. health officials on Tuesday approved the nation’s first gene therapy for an inherited disease, a treatment that improves the sight of patients with a rare form of blindness. It marks another major advance for the emerging field of genetic medicine.

The approval for Spark Therapeutics offers a life-changing intervention for a small group of patients with a vision-destroying genetic mutation and hope for many more people with other inherited diseases. The drugmaker said it will not disclose the price until next month, delaying debate about the affordability of a treatment analysts predict will be priced around $1 million.

The injection, called Luxturna, is the first gene therapy approved by the Food and Drug Administration in which a corrective gene is given directly to patients. The gene mutation interferes with the production of an enzyme needed for normal vision.

Patients who got the treatment have described seeing snow, stars or the moon for the first time.

“One of the best things I’ve ever seen since surgery are the stars. I never knew that they were little dots that twinkled,” said Mistie Lovelace of Kentucky, one of several patients who urged the FDA to approve the therapy at a public hearing in October.

Patients with the condition generally start losing their sight before 18, almost always progressing to total blindness. The defective gene that causes the disease can be passed down for generations undetected before suddenly appearing when a child inherits a copy from both parents. Only a few thousand people in the U.S. are thought to have the condition.