The U.S. Food and Drug Administration (FDA) on Tuesday approved the first gene therapy for an inherited disease. The injection, called Luxturna, treats patients with a rare form of blindness caused by genetic mutation. Drugmaker Spark Therapeutics said it would not disclose the price until January, but analysts predict the therapy will cost around $1 million.
People with the rare condition usually start to lose their eyesight before age 18 and almost always progress to total blindness. The therapy would involve two injections—one for each eye—that replace the defective gene with a corrective gene. A patient would need just one treatment, which some observers say explains the high price tag of $500,000 per eye. Patients enrolled in a key study of the therapy haven’t seen their vision deteriorate, some for as long as four years. “All the data we have today suggests it’s long-lasting, if not lifelong,” said Spark CEO Jeffrey Marrazzo.
Given FDA approval and strong study results, experts say they expect U.S. insurers to cover the treatment. —Kiley Crossland