Ciltacabtagene autoleucel
Ciltacabtagene autoleucel is a one-time BCMA CAR-T for myeloma that, in CARTITUDE-4, cut the risk of death by about 45% compared with standard regimens.
Approved 2022 (≥4 lines) and 2024 (≥1 prior line, lenalidomide-refractory; CARTITUDE-4 with OS benefit HR 0.55). A third of patients in CARTITUDE-1 remain progression-free at 5 years without maintenance. Delayed neurotoxicity (parkinsonism) and secondary malignancies are rare but notable. Legend Biotech/Johnson & Johnson.
1.Patient's T cells are collected by leukapheresis
- Route
- Single IV infusion after lymphodepletion
- Schedule
- 0.5–1.0 × 10⁶ CAR+ viable T cells/kg (max 1 × 10⁸); cyclophosphamide 300 mg/m² + fludarabine 30 mg/m² days −5 to −3
- Dose modifications
- Delay infusion for active infection; tocilizumab and steroids for CRS; anakinra for refractory
- Monitoring
- Daily monitoring for 10 days for CRS/ICANS; delayed neurotoxicity (parkinsonism) for months; blood counts and IgG; secondary malignancy surveillance for 15 years
Source: US prescribing information (DailyMed). Doses are for orientation; the current label governs.
- Medicare
- Part B (clinician-administered)NCD 110.24
Autologous CAR-T covered nationally under NCD 110.24 for FDA-labelled indications at facilities enrolled in the FDA REMS. Inpatient administration is paid under Part A (MS-DRG 018); outpatient administration is a Part B drug. The product itself is bundled into the facility payment. HCPCS Q2056.
- Commercial insurance
- covered with prior authorisation
Covered with prior authorisation and usually a single-case agreement with a certified treatment centre; many plans restrict to centres of excellence and require documentation of prior lines of therapy.
Sources: Medicare.gov: Chemotherapy · Medicare.gov: Prescription drugs (outpatient, Part B) · CMS NCD 110.24: Chimeric antigen receptor (CAR) T-cell therapy · Medicare.gov: Inpatient hospital care (Part A). Not medical or financial advice; verify with your plan.
- Notes
- Licensed 2022; NICE appraisals for later-line and CARTITUDE-4 earlier-line myeloma 2024-26. Check NICE.
Sources: NICE search: ciltacabtagene autoleucel. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
- Dec 2019DesignationUS
Breakthrough Therapy designation source
- 28 Feb 2022ApprovalUS
Relapsed/refractory myeloma after ≥4 lines (CARTITUDE-1) source
- 19 Jan 2024Label changeUS
Class boxed warning for T-cell malignancies source
- 5 Apr 2024ApprovalUS
Relapsed myeloma after ≥1 prior line, lenalidomide-refractory (CARTITUDE-4) source
- Jun 2025Label changeUS
REMS requirements removed for CAR-T class source
Approvals
| Region | Year | Indication |
|---|---|---|
| US | 2022 | Relapsed/refractory myeloma ≥4 lines |
| US | 2024 | Relapsed myeloma after ≥1 line, lenalidomide-refractory |
| Adverse event | Any grade | Grade 3+ |
|---|---|---|
| Cytokine release syndrome | 78% | 3% |
| Neutropenia (grade 3+) | — | 95% |
| Hypogammaglobulinaemia | 94% | 9% |
| Infections (grade 3+) | — | 24% |
| ICANS | 7% | 0.5% |
| Parkinsonism / movement disorders | 1% | 0% |
| Secondary haematological malignancies | 3% | — |
CARTITUDE-4, n=188. Rates read from the US prescribing information. Blank cells mean the figure was not sourced, not that it is zero.
| Country | Reimbursement | List price | Assistance |
|---|---|---|---|
| United States | Medicare Part B (physician-administered); commercial plans per formulary | $465,000 per infusion (list price at launch, 2022; 2026 list higher) | janssencarepath.com |
| United Kingdom | NICE: recommended for relapsed myeloma after ≥1 line, lenalidomide-refractory (2025) | not disclosed | — |
List prices are manufacturer or Medicare figures where publicly disclosed; net prices after rebates are usually lower. Reimbursement changes; check the payer.
Trials
topRecruiting now (live from ClinicalTrials.gov)
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CARTITUDE-4 is the first randomised trial to show that a CAR-T improves survival in myeloma, and it moved cilta-cel into second-line use (FDA approval 2024). For patients whose disease returns after first-line lenalidomide, a one-off cell therapy now competes with continuous drug combinations. Capacity, cost and the need for bridging therapy still limit who actually receives it.
CARTITUDE-1 showed that a single CAR-T infusion can put late-stage myeloma into deep, multi-year remission, leading to FDA approval of cilta-cel in 2022 for heavily pretreated disease. It set the efficacy bar for BCMA-directed therapy and motivated moving CAR-T earlier (CARTITUDE-4). Late neurological toxicity and secondary malignancies remain the safety questions.
Latest papers
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