Janssen No Longer Seeking Approval for Carvykti in the UK


Patients in the UK with r/r multiple myeloma may still be able to access the CAR-T through ongoing clinical trials, which are not impacted by the decision.

Janssen is no longer seeking the approval of ciltacabtagene autoleucel (cilta-cel; Carvykti), a chimeric antigen receptor T-cell (CAR-T) therapy for the treatment of relapsed/refractory (r/r) multiple myeloma (MM), by the UK’s National Institute of Health and Care Excellence (NICE), according to the charity group Myeloma UK.1 Carvykti, which was approved in the United States in 2022, was in the process of being evaluated by NICE.1,2

“Janssen agreed with NICE to withdraw the assessment of cilta-cel in the UK for the treatment of heavily pre-treated patients with multiple myeloma,” Brian Kenney, the global therapeutic area and cross-sector oncology communication leader for Janssen R&D and Johnson & Johnson External Innovation, said in a statement issued to CGTLive. “Despite our efforts and commitment to advancing this cell therapy for patients and physicians, we are not currently in a position to progress through the NICE appraisal process.”

According to Myeloma UK, the withdrawal was not related to any safety or efficacy concerns. Instead, the decision was likely related to production issues which have affected the ability of manufactures to meet demand for CAR-T therapies, which are more complex to produce than many other types of cancer treatments. The group noted that manufacturing issues have affected the availability of CAR-T therapies developed by other companies, including Bristol Myers Squibb’s ide-cel.

“While the withdrawal means that patients will not be able to access cilta-cel through the National Health Service at this time, the clinical trial program in the UK is not impacted by this development,” Kenney added. “We recognize the high unmet need of patients with multiple myeloma and remain committed to bringing CAR-T therapy to the multiple myeloma community.”

Carvykti’s approval in the United States was based on results of the now-completed phase 1b/2 CARTITUDE-1 clinical trial (NCT03548207). In CARTITUDE-1, patients treated with Carvykti achieved an objective response rateof 98% (95% CI, 92.7-99.7) and a stringent complete response rate of 78% (95% CI, 68.8-86.1). The median duration of response was 21.8 months at a median of 18 monthsof follow-up.3

Carvykti continues to be evaluated in additional ongoing clinical trials. In cohort B of the phase 2 CARTITUDE-2 clinical trial (NCT04133636), patients who had experienced early relapse after initial treatment for MM achieved a 100% overall response rate in data reported at the 2022 American Society of Clinical Oncology (ASCO) meeting, held June 3-7, 2022, both virtually and in Chicago, Illinois.4 More recently, in January 2023, Janssen announced that the phase 3 CARTITUDE-4 clinical trial (NCT04181827), which is evaluating Carvykti against standard of care regimens in the treatment of r/r MM, demonstrated a significant improvement in progression-free survival, the study's primary end point, over pomalidomide, bortezomib, and dexamethasone; and daratumumab, pomalidomide, and dexamethasone.5 In accordance with an independent data monitoring committee recommendation following the study meeting this end point, Janssen and Legend Biotech, which is codeveloping Carvykti, announced that CARTITUDE-4 will be unblinded.

1. Pharmaceutical company halts access to CAR-T cell treatment for UK myeloma patients. News release. Myeloma UK. March 13, 2023. Accessed March 17, 2023. https://www.myeloma.org.uk/news/pharmaceutical-company-halts-access-to-car-t-cell-treatment-for-uk-myeloma-patients/
2. U.S. FDA approves CARVYKTI™ (ciltacabtagene autoleucel), Janssen’s first cell therapy, a BCMA-directed CAR-T immunotherapy for the treatment of patients with relapsed or refractory multiple myeloma. News release. Janssen. February 28, 2022. Accessed March 17, 2023. https://bit.ly/35yWwjv
3. Carvykti (ciltacabtagene autoleucel) suspension for intravenous infusion. Prescribing information. Janssen Biotech and Legend Biotech Corporation; 2022. Accessed March 17, 2023. 2022.
4. van de Donk NWCJ, Agha ME, Cohen AD, et al. Biological correlative analyses and updated clinical data of ciltacabtagene autoleucel (cilta-cel), a BCMA-directed CAR-T cell therapy, in patients with multiple myeloma (MM) and early relapse after initial therapy: CARTITUDE-2, cohort B. Presented at: 2022 ASCO Annual Meeting, June 3-7, 2022. Poster #453.
5. Janssen announces unblinding of phase 3 CARTITUDE-4 study of CARVYKTI (cilta-cel) as primary endpoint met in treatment of patients with relapsed and refractory multiple myeloma. News release. The Janssen Pharmaceutical Companies of Johnson & Johnson. January 27, 2023. Accessed March 17, 2023. https://www.jnj.com/janssen-announces-unblinding-of-phase-3-cartitude-4-study-of-carvykti-cilta-cel-as-primary-endpoint-met-in-treatment-of-patients-with-relapsed-and-refractory-multiple-myeloma
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