Patients in focus

Just as FDA was wrapping up the inaugural meeting of its Patient Engagement Advisory Committee, another agency panel unanimously endorsed a gene therapy for a rare vision disorder based in large part on the testimony of patients, illustrating a continuing increase in the role of patient perspectives in regulatory decision-making.

On Oct. 12, FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted 16-0 that the benefit-risk profile of Luxturna voretigene neparvovec from Spark Therapeutics Inc. supported approval to treat a biallelic RPE65 mutation-associated retinal dystrophy...