FDA has granted regenerative medicine advanced therapy (RMAT) and fast track designations to Allogene Therapeutics for cemacabtagene ansegedleucel (cema-cel), an investigational allogeneic chimeric antigen receptor T cell (CAR-T) therapy, as a first-line (1L) consolidation treatment for patients with large B-cell lymphoma (LBCL) who test positive for minimal residual disease (MRD) after completing initial chemoimmunotherapy.1
"[FDA's] decision to grant both RMAT and [fast track] designations provides additional validation for the strategy we defined with the ALPHA3 trial and strengthens our ability to work closely with the agency on an efficient path to advance cema-cel as a first-line consolidation therapy for large B-cell lymphoma," said Zachary Roberts, MD, PhD, president and chief executive officer of Allogene Therapeutics, in a company press release.1
Key facts
- FDA grants RMAT and fast track designations to cema-cel
- Indication: 1L LBCL consolidation in MRD-positive patients
- 58.3% MRD negativity vs. 16.7% with observation (ALPHA3 interim data)
- 97.7% median ctDNA decrease vs. 26.6% increase, observation arm
- No treatment-related SAEs, no hospitalizations reported
- Cema-cel licensed from Cellectis via Servier; Allogene holds US, EU, and UK rights
What supported the designations?
FDA granted RMAT status following review of an interim futility analysis from the ongoing pivotal ALPHA3 trial, which is evaluating cema-cel against observation, the current standard of care, in 1L LBCL patients at high risk of relapse. At the protocol-defined data cutoff, 58.3% (7 of 12) of patients in the cema-cel arm achieved MRD negativity, compared with 16.7% (2 of 12) in the observation arm, a 41.6 percentage-point absolute difference.
The trial uses Natera's CLARITY MRD assay to identify patients in remission who remain at risk of relapse following 1L therapy. At Day 45, patients in the cema-cel arm also showed a 97.7% median decrease in plasma circulating tumor DNA (ctDNA), compared with a 26.6% median increase in the observation arm. Allogene noted that published literature and cross-study benchmarks suggest MRD-clearance differences of 25% to 30% may translate into clinically meaningful improvement by study completion.1
How did cema-cel perform on safety?
As of the data cutoff, cema-cel was well tolerated, with no treatment-related serious adverse events reported. There were no cases of cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome, graft-versus-host disease, or high-grade infections, and no patients required tocilizumab or steroids for toxicity management. Most patients were treated in the outpatient setting, and none were hospitalized for treatment-related adverse events, representing a profile that compares favorably with broader CAR-T experience, in which hospitalization for toxicity management remains common.1