Safety, efficacy consistent in Luxturna post-authorization study
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Interim safety data from a post-authorization study of Luxturna in patients with genetic retinal diseases were consistent with clinical trial data, according to a presentation at the American Society of Retina Specialists annual meeting.
Aaron Nagiel, MD, PhD, said the post-authorization safety study was designed to collect long-term safety information in patients treated with Luxturna (voretigene neparvovec-rzyl, Spark Therapeutics).
“Voretigene neparvovec is really a success story in gene therapy,” he said. “It’s the first FDA-approved AAV-based gene therapy.”
Researchers collected information on adverse events and serious adverse events, pregnancy outcomes and ophthalmic exams if available.
The study, an interim analysis up to 3 years, included data from 87 patients; 89.7% had a clinical diagnosis of Leber congenital amaurosis.
Nagiel said the adverse events were mostly mild and similar to what was seen in the clinical trials. Similarly, efficacy results were consistent with what was reported in the clinical trials.
However, he said there have been signals since approval related to chorioretinal atrophy. Out of 87 patients, eight experienced retinotomy touchdown only, which implies direct trauma likely from the surgical procedure, and 16 experienced the more concerning non-exclusively retinotomy touchdown.
Nearly 88% of patients with chorioretinal atrophy received voretigene neparvovec in both eyes and had atrophy in both eyes, but visual outcomes have been positive despite the atrophy.
“There were some signals that need to be followed up further in prospective studies,” Nagiel said. “One was a history of myopia, and the other was a delivery method using the automated foot pedal.”
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Nagiel A, et al. A multicenter post-authorization safety study of patients treated with voretigene neparvovec-rzyl gene therapy in the United States: Interim analysis at three years. Presented at: American Society of Retina Specialists annual meeting; July 17-20, 2024; Stockholm.
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