European Commission Approves Janssen and Legend Biotech’s Carvykti for Expanded Indication in Earlier Line Multiple Myeloma


The EC’s decision comes several weeks after the FDA approved cilta-cel for a similar expanded indication.

The European Commission (EC) has approved Janssen’s and Legend Biotech's ciltacabtagene autoleucel (cilta-cel; marketed as Carvykti) for an expanded indication in adult patients with relapsed/refractory multiple myeloma (MM) who have been treated with at least 1 prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent, showed disease progression on their last therapy, and have disease that is lenalidomide-refractory.1

The EC’s decision was based on data from the CARTITUDE-4 clinical trial (NCT04181827), a phase 3, randomized, open-label study in patients who received 1 to 3 prior lines of therapy for MM. CARTITUDE-4, which is still ongoing, is evaluating cilta-cel against standard of care (SOC) therapy, which consists of pomalidomide, bortezomib, and dexamethasone (PVd) or daratumumab, pomalidomide, and dexamethasone (DPd).

“The European Commission’s approval of CARVYKTI has the potential to transform the treatment paradigm for patients battling MM by bringing our novel therapy to them earlier in the course of this incurable disease,” Ying Huang, PhD, the chief executive officer of Legend Biotech, said in a statement.1 “This approval is a testament to our innovative science and galvanizes our efforts to provide new options that will improve outcomes for patients and give hope to them and their families.”

In CARTITUDE-4 data presented at the American Society of Clinical Oncology (ASCO) 2023 Annual Meeting, held June 2-6, in Chicago, Illinois, it was found that patients in the cilta-cel arm had a 12-month progression-free survival (PFS) rate of 76%, versus 49% in the SOC arm.2 Cilta-cel improved PFS in patients that had 1 or 2-3 prior lines of treatment. Patients with only 1 prior therapy may have experienced a stronger treatment benefit, but it was noted at the time that more data would be needed to confirm this trend.

Cilta-cel was previously approved by the FDA for an expanded indication in the second-line treatment of MM on April 6, 2024.3 The expanded FDA indication differs slightly, covering adult patients with relapsed and lenalidomide-refractory MM who have been treated with at least 1 prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent.

Cilta-cel was originally given conditional marketing authorization by the EC in May 2022.1 The indication covered by that decision included adult patients with relapsed/refractory MM who had been treated with at least 3 prior therapies, which were required to have included a proteasome inhibitor, an immunomodulatory agent, and an antiCD38 antibody, with disease progression having occurred while on the last treatment. The Type II variation application leading to the new expanded indication received a positive opinion from the Committee for Medicinal Products for Human Use of the European Medicines Agency in February 2024.4

1. Carvykti® (ciltacabtagene autoleucel) approved by the European commission for second-line treatment of patients with relapsed and refractory multiple myeloma. News release. Legend Biotech Corporation. April 22, 2024. Accessed April 23, 2024.
2. Dhakal B, Yong K, Harrison SJ, et al. First phase 3 results from CARTITUDE-4: Cilta-cel versus standard of care (PVd or DPd) in lenalidomide-refractory multiple myeloma. Presented at: ASCO 2023 Annual Meeting; June 2-6; Chicago, Illinois. Abstract #LBA106
3. CARVYKTI® is the first and only BCMA-targeted treatment approved by the U.S. FDA for patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy. News release. Johnson & Johnson. April 6, 2024. Accessed April 6, 2024.
4. Legend Biotech announces positive CHMP opinion for Carvykti®(ciltacabtagene autoleucel) for the treatment of patients with relapsed and lenalidomide refractory multiple myeloma in earlier lines of therapy. News release. Legend Biotech Corporation. April 22, 2024. Accessed April 23, 2024.
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