FDA approves gene therapy for a rare form of blindness

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.

The approval for Philadelphia-based Spark Therapeutics offers a lifechanging 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. Analysts predict it will be priced at about $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 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 appearing when a child inherits a copy from both parents. Only a few thousand people in the country are thought to have the condition.

Luxturna is delivered via two injections, one for each eye, that replace the defective gene that prevents the retina, tissue at the back of the eye, from converting light into electronic signals sent to the brain.

The FDA has approved three gene therapies since August. The previous two are treatments for forms of blood cancer. Novartis’ Kymriah costs $475,000; Gilead Sciences’ Yescarta is priced at $373,000 per treatment.

TOBIAS SCHWARZ/GETTY-AFP Mourners mark on Tuesday the 2016 truck attack at a Christmas market in Berlin. The attacker, a Tunisian asylum-seeker, killed 12 and hurt 70. He was killed days later in Italy.