The FDA has accepted a marketing application for Johnson & Johnson and partner Legend Biotech's BCMA-directed CAR-T therapy ciltacabtagene autoleucel (cilta-cel) for priority review as a potential treatment of adults with relapsed and/or refractory multiple myeloma, the latter company announced Wednesday. The US regulator is expected to make its decision by November 29.
The submission is backed by the pivotal Phase Ib/II CARTITUDE-1 study , in which cilta-cel produced an overall response rate of 97% and a stringent response rate of 67% after a median 12.4 months of follow-up time, according to data presented last December at the American Society of Hematology (ASH) meeting. The ongoing trial involves 126 patients with relapsed and/or refractory multiple myeloma who have received a proteasome inhibitor, an immunomodulatory drug and an anti-CD38 antibody as part of previous therapy, and who also experienced disease progression within 12 months of starting the most recent therapy.
"Cilta-cel has shown great promise in the treatment of patients with heavily pretreated multiple myeloma according to study findings reported to date," said Legend CEO Ying Huang, adding that "today's priority review designation marks another significant milestone for this cell therapy."
Meanwhile, safety data reported at last year's ASH meeting also revealed that most CARTITUDE-1 participants experienced cytokine release syndrome (CRS), with 3% of the cases classified as grade 3 and 1% each at grade 4 and grade 5. In addition, grade 3 or higher neurotoxicity was observed in 10% of patients, including 16 cases of immune effector cell-associated neurotoxicity syndrome (ICANS). There were also six deaths due to adverse events related to treatment, including sepsis, CRS and neurotoxicity. Updated longer term follow-up safety and efficacy data are due to be presented at the upcoming American Society of Clinical Oncology (ASCO) and European Hematology Association (EHA) conferences next month.
The FDA recently approved  Bristol Myers Squibb and bluebird bio's BCMA-targeting CAR-T therapy Abecma (idecabtagene vicleucel) as a later-line treatment for multiple myeloma. For related analysis, see ViewPoints: FDA filing sets up future CAR-T showdown in multiple myeloma . See also KOL Views Q&A: Leading MM expert confident anti-BCMA CAR-Ts like Abecma will move up from 4L .
Meanwhile, Legend submitted a filing for cilta-cel, also known as JNJ-4528 and LCAR-B38M, to European regulators late last month. The submission follows an accelerated assessment granted by the European Medicines Agency's Committee for Medicinal Products for Human Use based on the CARTITUDE-1 results.