The FDA announced Tuesday the approval of Spark Therapeutics' gene therapy Luxturna (voretigene neparvovec-rzyl) for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. The agency noted that Luxturna is the first directly administered gene therapy authorised in the US that targets a disease caused by mutations in a specific gene. FDA Commissioner Scott Gottlieb remarked "today's approval marks another first in the field of gene therapy…and this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases."
Approval of Luxturna follows a unanimous vote by an FDA advisory panel in October backing clearance of the drug. In study data published earlier this year, the one-time gene therapy was demonstrated to improve functional vision, light sensitivity and visual field in study participants with RPE65-mediated inherited retinal disease.
Spark said it will not disclose the price of Luxturna until January but Goldman Sachs analyst Salveen Richter expects the drug will carry a price of $500 000 per injection, or $1 million for both eyes. Meanwhile, the drugmaker recently suggested that there is a case for valuing the therapy at more than $1 million annually (for related analysis, read Spotlight On: Gene therapy companies open up about pricing strategies).
Luxturna, which is expected to be available in some US centers late in the first quarter of next year, was previously granted priority review, breakthrough therapy and orphan drug designations, while Spark will be eligible for a rare paediatric disease priority review voucher based on the clearance of the drug. The treatment remains under review in Europe.
Luxturna is the third gene therapy approved by the FDA, following Novartis' CAR-T therapy Kymriah (tisagenlecleucel) for certain children and young adults with acute lymphoblastic leukaemia and Gilead Sciences' CAR-T therapy Yescarta (axicabtagene ciloleucel) for certain patients with large B-cell lymphoma.
For additional analysis, see ViewPoints: Panel augurs well for Spark’s Luxturna – if commercial intricacies are managed.
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