An FDA advisory panel on Wednesday voted unanimously in favour of recommending approval for Novartis' experimental CAR-T treatment tisagenlecleucel-T, also known as CTL019, for patients aged three to 25 years with relapsed/refractory B-cell acute lymphoblastic leukaemia (ALL). If cleared by the FDA, which is expected to make its decision by the end of September, tisagenlecleucel-T would be the first such therapy approved in the US.
A report earlier this week suggested that panellists would focus on the treatment's short-term and long-term safety given the efficacy already demonstrated in clinical trials. Data from the Phase II ELIANA trial unveiled last year showed that 41 of 50 patients achieved complete remission or complete remission with incomplete blood count recovery at three months post tisagenlecleucel-T infusion. However, 48 percent of patients developed grade 3 or 4 cytokine release syndrome, but Novartis noted that none of the cases was fatal.
Analysts have forecast peak sales of at least $1 billion for tisagenlecleucel-T, which Novartis is also evaluating for other indications. In June, the Swiss drugmaker released positive results from the mid-stage JULIET study for the therapy in the treatment of adults with relapsed or refractory diffuse large B-cell lymphoma.
Commenting on the advisory panel vote, Raymond James analyst Reni Benjamin remarked "in the last five years there have been a significant number of cell therapy companies that have gone public or gotten investment in hopes of moving this type of therapy forward…[but] this is our first glimpse from a commercial and regulatory perspective about how the FDA is thinking about this space."
Additionally, Jefferies estimated that tisagenlecleucel-T, if approved, will provide "an important sustainable revenue stream" of about 65 million pounds ($83.7 million) to 75 million pounds ($96.2 million) annually for Oxford BioMedica. Earlier this month, Novartis and Oxford BioMedica expanded their existing partnership regarding the supply of lentiviral vectors used in tisagenlecleucel-T and other CAR-T treatments.
Earlier this year, Juno Therapeutics ended development of its CAR-T drug JCAR015 in adults with relapsed or refractory ALL following safety issues observed in the Phase II ROCKET trial, including five patient deaths due to cerebral oedema. Meanwhile, the FDA is slated to rule on Kite Pharma's submission for the investigational CAR-T treatment axicabtagene ciloleucel by the end of November. The company is seeking to have the therapy approved for refractory aggressive non-Hodgkin lymphoma.
For related analysis, see ViewPoints: Novartis wins landmark recommendation for CAR-T therapy.
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