Abecma wins CHMP’s thumbs up for use in earlier treatment lines
Therapy already approved in Europe for relapsed and hard-to-treat myeloma
Bristol Myers Squibb has received a positive opinion from an arm of the European Medicines Agency (EMA) on the use of its CAR T-cell therapy Abecma (idecabtagene vicleucel) in earlier lines of treatment for relapsed and hard-to-treat multiple myeloma.
Abecma already is approved in the European Union to treat adults whose multiple myeloma has relapsed, or come back, and is refractory — difficult to treat and typically not responding to therapy — despite having been through at least three lines of treatment. Specifically, patients will already have tried an immunomodulator, a proteasome inhibitor, and a CD38 inhibitor.
Now, the developer is seeking an expanded approval for adults who have received at least two lines of treatment — similar to an application already given the green light in Japan, where Abecma late last year became the first CAR T-cell therapy to be approved for use in earlier lines of treatment for myeloma. A similar application is under review in the U.S.
The positive opinion was issued by the Committee for Medicinal Products for Human Use (CHMP), an arm of the EMA. With it in hand, the company can expect the European Commission, which has the authority to issue an approval in the European Union, to make a final decision within about two months.
“We look forward to working with the European Commission with the shared goal of delivering innovative treatment options to more patients with continued unmet need,” Anne Kerber, MD, senior vice president and head of late clinical development, hematology, oncology, and cell therapy at Bristol Myers Squibb, said in a press release.
Similar application approved in Japan, under review in US
Abecma is a CAR T-cell therapy, a type of immunotherapy that uses the body’s own immune system to target cancer cells. It involves collecting a patient’s T-cells and reprogramming them in the lab in a way that they can recognize a protein called B-cell maturation antigen, or BCMA. This protein is most abundant on the surface of myeloma cells.
When the reprogrammed T-cells are infused back to the patient, they travel in the bloodstream throughout the body. As they encounter a BCMA protein, they hook onto it and destroy the carrier myeloma cell.
CHMP’s positive opinion was based on data from a Phase 3 clinical study called KarMMa-3 (NCT03651128), where Abecma outperformed standard regimens in adults with relapsed and refractory multiple myeloma who had received 2-4 lines of treatment.
“This positive CHMP opinion represents an important step toward bringing our potentially transformative first-in-class anti-BCMA CAR-T cell therapy, Abecma, to more patients earlier in the multiple myeloma treatment paradigm to improve outcomes,” Kerber said.
The KarMMa-3 study included 386 adults with RRMM who had tried at least three treatments — an immunomodulator, a proteasome inhibitor, and the CD38 inhibitor Darzalex (daratumumab). As part of the study, participants were randomly assigned to receive either Abecma or one of five standard regimens.
After a median of 30.9 months, or about 2.5 years, Abecma had cut the risk of the disease getting worse or causing death by about half compared with the standard regimens. Median progression-free survival, a measure of the time a patient lives without the cancer progressing, also was significantly longer for Abecma (13.8 vs. 4.4 months).
This positive CHMP opinion represents an important step toward bringing our potentially transformative first-in-class anti-BCMA CAR-T cell therapy, Abecma, to more patients earlier in the multiple myeloma treatment paradigm to improve outcomes.
Those data, presented at the American Society of Hematology’s annual meeting in late 2023, also showed that most patients (71%) responded to treatment with Abecma, with 44% achieving a complete or stringent complete response, meaning no detectable cancer.
In contrast, fewer than half (41%) responded to standard regimens, with 5% achieving a complete or stringent complete response with those treatments.
Of the 225 patients who received Abecma, the vast majority — 197 or 88% — experienced cytokine release syndrome, a sudden, heightened inflammatory response, with mostly low-grade events. Neurotoxicity occurred in 34 patients (15%), with seven (3%) experiencing a serious or life-threatening event.
Should the European Commission issue a final decision favorable to Abecma’s expanded approval, it would be applicable to all member countries of the EU, plus Iceland, Norway, and Liechtenstein.