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BioWorld - Thursday, April 30, 2026
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Home » CBER head Peter Marks weighs in on cell, gene therapy pricing debate
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CBER head Peter Marks weighs in on cell, gene therapy pricing debate

May 16, 2018
By Cormac Sheridan
DUBLIN – Last week Philadelphia-based Spark Therapeutics Inc. reported $2.4 million revenue for the debut quarter of Luxturna (voretigene neparvovec-rzyl), its gene therapy for an inherited form of vision loss, which gained FDA approval last year. Novartis AG also recently reported its first quarterly sales figure for Kymriah (tisagenlecleucel). The chimeric antigen receptor T-cell (CAR-T) therapy took in $12 million in the first quarter, its second on the market since its approval last August for children and young adults with B-cell precursor acute lymphoblastic leukemia (ALL). Gilead Sciences Inc. reported Q1 sales of $40 million for Yescarta (axicabtagene ciloleucel), the CD19-directed CAR-T therapy approved for diffuse large B-cell lymphoma, following sales of $7 million in Q4 2017, its first quarter on the market. Although modest, the numbers demonstrate that the new era of cell and gene therapy has – after several false dawns in Europe – finally arrived.
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