FDA grants regenerative medicine advanced therapy and fast-track designation to equecabtagene autoleucel for RRMM

On February 12, 2023, it was announced that equecabtagene autoleucel (previously known as CT103A), a BCMA-directed CAR T-cell therapy received both regenerative medicine advanced therapy and fast-track designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with relapsed/refractory (R/R) multiple myeloma (MM). This follows the orphan drug designation in February 2022 and investigational new drug approval by the U.S FDA in December 2022.¹

The Multiple Myeloma Hub previously reported the phase I clinical data of equecabtagene autoleucel in patients with RRMM which demonstrated increased persistency and efficacious responses, including those treated with prior murine BCMA CAR T-cell treatment. The updated phase Ib/II study results of the ongoing FUMANBA-1 trial (NCT05066646) were presented by Li² during the European Hematology Association (EHA) 2022 Congress. Key results at the data cut off for the 79 evaluable patients with RRMM who were previously treated with at least three lines of therapy were as follows:

• An overall response rate of 94.9%, with 68.4% achieving a complete response rate or higher.
• A median time to response of 16 days.
• Observed responses in patients treated with prior non-BCMA CAR T-cell therapy.
• MRD-negativity in 92.4% overall.
• All cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome incidences were Grade 1 and 2 and none were Grade 3 or higher.
• Equecabtagene autoleucel demonstrated prolonged persistency and low immunogenicity.

Additional ongoing clinical trials with equecabtagene autoleucel are:

• A phase I study combined with selinexor in patients with extramedullary RRMM and had at least three prior lines of therapy (NCT05201118).
• A phase I study in patients with high-risk newly diagnosed MM (FUMANBA-2, NCT05181501), defined as Revised International Staging System (R-ISS) Stage 3, double-hit, or triple-hit.