FDA panel approves a gene therapy for blindness

A gene therapy for a genetically inherited form of blindness got approval from an FDA panel Thursday and could get final agency approval for market by January. If the FDA signs off on it, it will be the first-ever genetic treatment for a genetically inherited disease to be available to U.S. consumers.
By Miriam Griffin | Oct 16, 2017
A drug-administered gene therapy to treat an inherited form of blindness got unanimous approval Thursday from a U.S. Food and Drug Administration panel. The agency will make a final decision on whether to approve the drug by January, according to researchers who said that it will be the first time in the FDA's history that it has approved a gene therapy to treat a genetically inherited disease.

The drug, Luxturna, is a product of Philadelphia-based pharmaceutical company Spark Therapeutics and is made for patients suffering from Leber congenital aumarosis, a blindness that is caused by a mutation of the RPE65 gene. The patients receive the drug via injection into their eyes, and it delivers a correct copy of the RPE65 gene to their retinal cells, which leads to healthy cells that function properly and enable working eyesight.

The FDA has approved one gene therapy so far: Kymriah, which treats a form of leukemia by transferring new genes to the cancer patients' cells. Two other drugs that use genes to treat genetically inherited diseases have received approval for market in Europe. But Jeff Marrazzo, CEO of Spark Therapeutics, noted that an FDA approval of Luxturna would make it the first time that U.S. consumers will have access to a gene therapy that treats a genetically inherited disease.

"This is what I believe medicine is going to be like for the next 20, 30, if not 50 years," he said. "I think this is the beginning of an age that is going to fundamentally change medicine."

In one study of 21 patients who underwent the procedure, 11 experienced "significant" vision improvement. No conclusive data is available on how long the improved vision lasts, however, or how much the treatment will cost.


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