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Patients in focus

How patient input helped FDA panel understand benefits of Spark gene therapy

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.

Luxturna is an adeno-associated virus (AAV) encoding the retinal pigment epithelium-specific protein 65kDA (RPE65) gene. If FDA follows the panel’s recommendation, Luxturna would be the first AAV gene therapy approved in the U.S.

Patient testimony at the panel was particularly critical in putting into perspective data on Spark’s novel primary endpoint, the multi-luminance mobility test (MLMT). The test assesses an individual’s ability to navigate an obstacle course at six different levels of light, from bright office-like

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