# FDA Approval Summary: Ciltacabtagene Autoleucel for Relapsed or Refractory Multiple Myeloma

Source: https://onco.cc/key-papers/paper-ciltacabtagene-autoleucel-multiple-myeloma-clin-cancer-res-2024/  
OnCo record `paper-ciltacabtagene-autoleucel-multiple-myeloma-clin-cancer-res-2024` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Phase 2 or 3 results paper on Ciltacabtagene autoleucel in Multiple myeloma, in Clinical Cancer Research (2024), one of the most cited Europe PMC records with Ciltacabtagene autoleucel in its title.

## Summary

In February 2022, the FDA approved ciltacabtagene autoleucel, a chimeric antigen receptor (CAR) T-cell therapy targeting the B-cell maturation antigen, for adult patients with relapsed/refractory multiple myeloma after ≥4 lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody. Approval was based on overall response rate (ORR), complete response (CR) rate, and duration of response (DoR) in 97 adult patients in a single-arm, open-label, multicenter phase 2 trial (CARTITUDE-1 [NCT03548207]). Patients received a single infusion of ciltacabtagene autoleucel, preceded by lymphodepleting chemotherapy. Of the 97 patients evaluable, ORR was 97.9% [95% confidence interval (CI), 92.7-99.7] with a stringent CR rate of 78.4% (95% CI, 68.8-86.1). After median follow-up of 18 months, the median DoR was 21.8 months (95% CI, 21.8-not estimable [NE]) in responders (PR or better) and NE (95% CI, 21.8 months-NE) in patients who achieved stringent CR. Serious adverse reactions occurred in 55% of the 97 patients evaluated for safety. Grade 3 or higher cytokine release syndrome (CRS) and neurologic toxicities occurred in 5% and 11% of the patients, respectively, leading to a Risk Evaluation and Mitigation Strategy. Neurologic toxicities included immune effector cell-associated neurologic syndrome, typically seen with CAR-T products, parkinsonism, peripheral neuropathy, cranial nerve palsies, and Guillain-Barré syndrome. One fatal case of hemophagocytic lymphohistiocytosis/macrophage activation syndrome occurred. Prolonged and recurrent grade 3 or 4 cytopenias occurred; a single patient required hematopoietic stem-cell rescue.

Indexed on Europe PMC as PubMed record 38713595 (DOI 10.1158/1078-0432.ccr-24-0378). Its title names Ciltacabtagene autoleucel and its text names Multiple myeloma; PubMed types it as a clinical trial report (Clinical Trial, Phase II, research-article, Multicenter Study). It was matched automatically to the idea "One CAR-T infusion instead of autologous transplant" and no figure has been checked by an editor.

## Fields

- Kind: Key paper
- Last checked: 2026-09-22
- Tags: europepmc-ingest
- Journal: Clinical Cancer Research
- Year: 2024
- DOI: 10.1158/1078-0432.ccr-24-0378
- Authors: Natrajan K, Kaushal M, George B, et al.
- What it means: One of the most cited trial reports Europe PMC returns for Ciltacabtagene autoleucel in Multiple myeloma, so it is a natural first reading for anyone weighing the idea it is linked from. The record was linked automatically by title and abstract; read the abstract above and the paper itself before relying on any figure.
- Caveats: Matched by Ciltacabtagene autoleucel in the title and Multiple myeloma in the title or abstract of the Europe PMC record; the summary reproduces the record's abstract and no figure has been verified against the full paper.; Chosen as a phase 2 or 3 report by the record's publication type or its own wording, not by reading the paper.

## Sources

- Clin Cancer Res 2024: https://doi.org/10.1158/1078-0432.ccr-24-0378
- PubMed: https://pubmed.ncbi.nlm.nih.gov/38713595/
- Europe PMC: https://europepmc.org/article/MED/38713595
- ClinicalTrials.gov NCT03548207: https://clinicaltrials.gov/study/NCT03548207

## Connected records

- trials: [CARTITUDE-1](https://onco.cc/trials/cartitude-1/)
- journals: [Clinical Cancer Research](https://onco.cc/journals/clinical-cancer-research/)
- ideas: [One CAR-T infusion instead of autologous transplant](https://onco.cc/ideas/idea-car-t-replaces-transplant/)

---
JSON: https://onco.cc/api/v1/entities/paper-ciltacabtagene-autoleucel-multiple-myeloma-clin-cancer-res-2024.json