Gene therapy for a rare form of blindness wins FDA approval
WASHINGTON — Federal health officials have 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 Tuesday 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 that analysts predict will be priced around $1 million.