Innovative personalised immunotherapy approved for multiple myeloma

The first BCMA-targeted CAR-T cell therapy for second-line treatment of multiple myeloma has been approved by the US Food and Drug Administration (FDA). CARVYKTI ^® (ciltacabtagene autoleucel; cilta-cel) is indicated for patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy, including a proteasome inhibitor (PI), and an immunomodulatory agent (IMiD), and are refractory to lenalidomide.

About the immunotherapy

Ciltacabtagene autoleucel is an autologous T-cell immunotherapy. When the cilta-cel CAR protein binds to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells, Legend Biotech explained.

“The expanded label of CARVYKTI has the potential to transform the treatment paradigm for multiple myeloma by providing patients and physicians with a personalised immunotherapy that can be used earlier in the treatment regimen,” commented Ying Huang, PhD, Chief Executive Officer of Legend Biotech.

Basis of the approval of the CAR-T therapy

FDA’s approval of the personalised treatment is based on positive results from the Phase III CARTITUDE-4 study.

The data demonstrated that CARVYKTI offered statistically significant improvement of progression-free survival compared to two standard of care treatment regimens: pomalidomide, bortezomib, and dexamethasone (PVd) or daratumumab, pomalidomide, and dexamethasone (DPd).

“This expanded FDA approval for cilta-cel will allow a wider patient population to access this novel therapy earlier in the course of their treatment,” according to Dr Surbhi Sidana, Assistant Professor of Medicine, Blood and Marrow Transplantation & Cellular Therapy at Stanford University School of Medicine.

The benefit of the cilta-cel infusion was observed when compared to standard of care continuous therapy in patients who relapsed after one to three prior treatments, Dr Sidana added, 

Legend Biotech entered into a license and collaboration agreement with Janssen Biotech, Inc. (Janssen), a Johnson & Johnson company in December 2017. The partnership deal focused on the development and commercialisation of the immunotherapy CAR-T cell therapy cilta-cel.

FDA approves Carvykti for patients with multiple myeloma