Spark Therapeutics' experimental gene therapy, Luxturna, improves vision in patients with a rare, inherited form of blindness, according to an initial review by Food and Drug Administration.
Here are four things to know.
1. Luxturna is a one-time gene therapy intended to treat about 1,000 to 2,000 Americans with the inherited retinal disease Leber congenital amaurosis.
2. The FDA acknowledged Luxturna's short-term efficacy, but said long-term clinical benefits are unknown, since no data exists on the gene therapy's efficacy after a year.
3. If approved, Luxturna would be the first gene therapy used to treat an inherited disease in the U.S.
4. Spark Therapeutics CEO Jeffrey Marrazzo declined to comment on the future price of the drug during an interview with The New York Times. However, he did say the company will consider the price of other rare disease drugs, such as treatments for Pompe disease and Hunter syndrome, when setting a price for Luxturna. Many of these drugs cost $300,000 to $600,000 annually.
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