On Thursday, a US Food and Drug Administration review panel gave their stamp of approval to an experimental gene therapy for a rare inherited form of blindness. If ultimately approved by the agency, the technique would be the first gene therapy for an inherited disease approved in the United States—and a landmark in the field of biomedicine.
The drug, Luxturna, from Spark Therapeutics, treats an inherited blindness condition known as Leber congenital aumaurosis—a condition triggered by mutations to the RPE65 gene. The drug works by delivering a correct copy of the RP65 gene to retinal cells, restoring a person’s ability to produce the deficient enzyme that causes the condition.
In a key study of 21 patients, 11 patients who underwent the experimental treatment experienced significant vision improvement. A total of 93 percent of participants experienced at least some improvement, with improvement measured by an ability to navigate obstacles under poor lighting conditions.
Earlier this week, a review committee published a document that found the therapy does improve vision, at least in the short-term, and met criteria for approval. But the review also noted that there is no long-term data on how the drug works over time.
On Thursday, the panel of outside experts gave the drug unanimous approval. The agency must now approve the drug by January.