FDA joins EU and Japan in deciding if Abecma may treat RRMM earlier

In US, CAR T-cell therapy now an option after 4 or more lines of treatment

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

Share this article:

Share article via email
An illustration showing a bell with the word

The U.S. Food and Drug Administration (FDA) has agreed to review an application that, if approved, would allow the cell therapy Abecma (idecabtagene vicleucel) to be given earlier to people with hard-to-treat multiple myeloma.

An FDA decision is expected by Dec. 16, according to Abecma’s developers Bristol Myers Squibb (BMS) and 2seventy bio.

“Our continued focus on bringing Abecma into earlier lines of treatment demonstrates our commitment to increasing treatment options and improving outcomes for patients living with multiple myeloma,” Anne Kerber, MD, senior vice president and head of cell therapy development at BMS, said in a joint company press release. “This FDA acceptance marks another step forward in our mission by bringing us closer to offering this potentially transformative, one-time CAR T treatment option to more patients.”

Similar applications are being reviewed by health authorities for the European Union and Japan.

Recommended Reading
A woman makes an announcement into a megaphone.

Elranatamab under FDA priority review for hard-to-treat myeloma

Applications supported by Phase 3 KarMMa-3 trial results

Abecma is a CAR T-cell therapy that involves collecting a patient’s immune T-cells, modifying them in a lab to make them better at killing myeloma cells, and infusing them back to the patient. The modified cells are equipped with a chimeric antigen receptor, or CAR, that targets BCMA, a protein present at high levels on the surface of myeloma cells.

In the U.S., Abecma currently is approved for adults with relapsed or refractory multiple myeloma (RRMM) who have received at least four prior therapy lines, or regimens, including at least one immunomodulatory drug, one proteasome inhibitor, and one CD38 inhibitor.

If the new application is approved by the FDA, this CAR T-cell treatment would be available as an earlier line of therapy to RRMM patients who have received these three classes of therapies — “the most commonly used standard treatments in multiple myeloma,” the release stated.

In the European Union and Japan, Abecma currently is approved for patients who’ve been given at least three prior therapies, including at least one of each of the three classes of medication mentioned above.

“These global regulatory acceptances of BMS’ applications represent important additional progress across three regions with significant patient need in triple-class exposed multiple myeloma,” said Adam Lenkowsky, senior vice president and head of major markets at BMS.

The applications are supported by findings from the Phase 3 KarMMa-3 clinical trial (NCT03651128).

The study enrolled 386 adult RRMM patients who failed to respond to the most recent of two to four treatment regimens, which had to include an immunomodulatory agent, a proteasome inhibitor, and the CD38 inhibitor Darzalex (daratumumab).

Participants were randomly assigned to be treated with Abecma or one of five standard combination therapies.

Results showed that Abecma was associated with a 51% lower risk of disease progression or death relative to standard therapy. On average, the 254 trial patients given the cell therapy went for more than a year without disease progression following treatment, compared with less than five months for those given standard treatment regimens.

Data also demonstrated that a significantly greater proportion of patients given Abecma responded to treatment, or had a reduction in cancer burden, than did standard treatment patients (71% vs. 42%).

Safety findings for the cell therapy were consistent with earlier trials. Serious and life-threatening inflammatory and neurological reactions were rare among Abecma-treated patients in this and other studies.

“Positive results from our Phase 3 KarMMa-3 study demonstrate a significant clinical benefit of Abecma across lines of care in triple-class exposed multiple myeloma,” said Steve Bernstein, MD, chief medical officer of 2seventy.

“The KarMMa-3 study has shown the clear clinical benefit of Abecma over existing standard of care regimens and, if approved, the potential for this anti-BCMA CAR T cell therapy to become a standard of care earlier in the treatment course for relapsed and refractory multiple myeloma,” Lenkowsky said.