Rolling Submission Begins for BLA on Cilta-Cel in R/R Multiple Myeloma

Results presented earlier this month at ASH showed a 97% overall response rate, and progression-free survival had not been reached.

Janssen Pharmaceutical on Monday began a rolling submission of a Biologics License Application (BLA) for the BCMA CAR-T therapy ciltacabtagene autoleucel (cilta-cel), which is being developed to treat patients who have had as many as 6 prior treatments for multiple myeloma

“We are committed to innovation in cell therapy and advancing the science of multiple myeloma to improve patients' lives,” Peter Lebowitz, MD, PhD, Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC, said in a statement.

“Today's milestone is the culmination of a remarkable clinical development effort and collaboration with Legend Biotech. We look forward to working with the FDA in their review of cilta-cel with the goal of bringing a highly-active, dual-binding BCMA CAR-T therapy to patients with relapsed and/or refractory multiple myeloma who are in need of new treatment options.”

In this next generation of chimeric antigen receptor (CAR) T-cell therapy, the CAR can target and recognize the B-cell maturation antigen (BCMA), and kill those multiple myeloma cells that have expressed this antigen. Cilta-cel contains a 4-1BB co-stimulatory domain and 2 BCMA-targeting single-domain antibodies with a preferential CD8+ T-cell expansion.

In clinical trials, the therapy is showing efficacy in patients who have been refractory for as many as 5 earlier treatments. Patients with multiple myeloma who are refractory after 3 or more lines of therapy typically have a poor prognosis, according to Deepu Madduri, MD, of the Tisch Cancer Institute at Mt. Sinai, New York, New York. Earlier this month, Madduri presented data on 97 patients who had received cilta-cel in the phase 1b/2 CARTITUDE-1 trial during the 62nd American Society of Hematology Annual Meeting and Exposition.

CARTITUDE-1 showed cilta-cel had an overall response rate of 97%, with a stringent response rate of 67%. Median duration of response and progression-free survival were not reached at the time of data cut-off for the presentation at ASH, after a median follow-up of 12.4 months.

According to a statement from Janssen, which his developing the therapy with Legend Biotech, FDA previously granted Breakthrough Therapy Designation for cilta-cel and has agreed to a rolling review of the BLA, which allows completed portions of the application to be submitted and reviewed on an ongoing basis.


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