FDA Approves Luxturna: Gene Therapy for Rare Form of Blindness

By: | January 15th, 2018


Image courtesy Spark Therapeutics

Gene therapy uses genes to treat or prevent disease instead of using drugs or surgery.

Recently, the FDA approved the first gene therapy for an inherited retinal disorder. The therapy is called Luxturna. The revolutionary treatment aims to cure a rare form of blindness affecting between 1,000 and 2,000 Americans.

The first-of-its-kind genetic treatment for a rare eye disease that causes blindness will cost $850,000

Luxturna is an injection, one for each eye, to replace a defective gene in the retinal tissue at the back of the eye that converts light into electrical signals to produce vision. The therapy will cost $425,000 per injection.

Spark Therapeutics, the company producing Luxturna, decided to lower the cost of the injectable treatment to $850,000 after an uproar from health insurers over the drug’s expected $1 million price tag.

The therapy is still among the most expensive genetic therapies in the world.

“We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients,” Spark Theraputics’ CEO Jeffrey Marrazzo said.

Nidhi Goyal

Nidhi is a gold medalist Post Graduate in Atmospheric and Oceanic Sciences.

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