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.
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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 per cent 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.