Abecma OK’d in US to take earlier in treatments for multiple myeloma

Patients in trial saw 51% reduced risk of disease progression and death

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by Andrea Lobo |

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The U.S. Food and Drug Administration (FDA) has expanded the indication of Abecma (idecabtagene vicleucel) to allow its use earlier in the therapy regimen for adults with difficult-to-treat multiple myeloma.

The CAR T-cell therapy, developed by Bristol Myers Squibb (BMS) and 2seventy bio, was already approved in the U.S. for people with relapsed or refractory myeloma (RRMM) who received at least four previous lines of therapy. Eligible patients had to be triple-class exposed, meaning they received at least one immunomodulatory drug, one proteasome inhibitor, and one CD38 inhibitor.

The increasing use of combination regimens as a single line of treatment means more patients become triple-class exposed earlier, with limited treatment options available for these patients.

The FDA’s label expansion now makes Abecma available for triple-class exposed patients given at least two prior lines of therapy if they relapsed or become resistant to the past treatment. The regulatory decision came about a month after an agency’s advisory committee voted in favor of the therapy’s expanded use.

Similar label extensions were cleared in the European Union, Japan, and Switzerland.

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“We are extremely pleased that Abecma will be available to many more patients in the U.S.,” Chip Baird, 2seventy bio’s CEO, said in a joint company press release. “This approval represents another important milestone for patients.”

Bryan Campbell, BMS’s senior vice president and head of commercial and cell therapy, said that “Abecma has demonstrated a progression-free survival benefit three times that of standard regimens in relapsed or refractory multiple myeloma, and we are now bringing the promise of cell therapy to patients earlier in their treatment journey.”

Multiple myeloma is a form of blood cancer caused by the uncontrolled production of plasma cells — immune B-cells that produce large amounts of antibodies — that lead to several plasma cell tumors inside and/or outside the bones.

Abecma is a cell therapy that involves collecting a patient’s T-cells, a type of immune cell, and engineering them in the lab to recognize the B-cell maturation antigen (BMCA) protein on myeloma cells. The modified T-cells are then infused back to the patient, where they will bind BCMA and launch an attack against myeloma cells.

Clinical trial results

The therapy’s label extensions were supported by results of the Phase 3 KarMMa-3 clinical trial (NCT03651128). The study recruited 386 adults with RRMM who’d received two to four treatment lines that made them triple-class exposed. They were randomly assigned to receive either Abecma or one of five standard treatment regimens.

On average, patients on Abecma lived without disease progression for more than a year (13.3 months ), compared with 4.4 months for those on standard regimens. This represented a 51% reduction in the risk of disease progression or death with Abecma.

Moreover, most Abecma-treated patients (71%) were classified as treatment responders, with 39% attaining a complete or stringent complete response, meaning no cancer was detectable. In turn, less than half (42%) of those on standard regimens achieved a response, and only 5% had a complete response or better.

Also, responses to Abecma treatment lasted a median of 14.8 months (more than a year), and for patients achieving at least a complete response, a median of 20 months (nearly two years).

Pooled data from Abecma trials has shown that although most treated patients had cytokine release syndrome (89%), a sudden type of immune response common with CAR T-cell therapies, most were not severe. Moreover, neurotoxicity occurred in 40% of Abecma-treated patients, yet it was mainly not severe.

“The results of the KarMMa-3 study are remarkable, especially given the historic outcomes with standard regimens for these patients with relapsed or refractory disease,” said Al-Ola A. Abdallah, MD, an associate professor at University of Kansas Medical Center and chair of the U.S. Myeloma Innovations Research Collaborative.

“These patients now have an opportunity to be treated at an earlier line of therapy with a potentially transformative therapy that offers significantly improved progression-free survival for this difficult-to-treat disease that has had no established treatment approach,” Abdallah added.