US-based Janssen Pharmaceutical reportedly decided to withdraw Carvykti (ciltacabtagene autoleucel; cilta-cel) from the UK market last week. The BCMA-targeted chimeric antigen receptor (CAR) T cell therapy, which is under development globally by Janssen in partnership with China-based Legend Biotech (NASDAQ: LEGN), was being assessed by the UK’s National Institute of Health and Care Excellence (NICE) for reimbursement within the National Health Service (NHS) when it was withdrawn by Janssen.
Reason Behind the Decision
The decision was publicized to UK cancer patients by the cancer charity Myeloma UK, which noted that the withdrawal reflected no safety or efficacy concerns. Instead, the charity speculated that a lack of manufacturing capability globally may have been the primary reason behind the decision not to supply the CAR-T to the UK market. Myeloma UK also highlighted that Bristol-Myers Squibb (BMS, NYSE: BMY) ended UK development of the first-in-class BCMA CAR-T Abecma (idecabtagene vicleucel) in 2020, citing similar manufacturing issues.
NICE’s Role and CAR-T Challenges
As the cost-effectiveness watchdog for the NHS, NICE determines whether drugs should be made freely available to patients. The agency’s strict adherence to a maximum GBP 20,000-GBP 30,000 per quality-adjusted life year (QALY) for a treatment to be considered cost-effective may represent a significant hurdle for the reimbursement of CAR-T therapies in the UK.
Janssen’s Statement and Future Development
Janssen released a statement from Brian Kenney, the global therapeutic area and cross-sector oncology communication leader for Janssen R&D and Johnson & Johnson External Innovation, noting: “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.” The decision not to continue with the NICE appraisal for NHS coverage of Carvykti will not affect clinical trials held in the UK. Carvykti won its first major market approvals last year, starting in the US in March and followed by the European Medicines Agency in May, indicated for heavily pre-treated patients with relapsed/refractory multiple myeloma (MM). Janssen continues to develop the drug for various indications related to MM.-Fineline Info & Tech
