FDA grants RMAT designation to ALLO-501 CAR-T for advanced large B-cell lymphoma
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The FDA granted regenerative medicine advanced therapy designation to ALLO-501, a chimeric antigen receptor T-cell therapy, for the treatment of adults with relapsed or refractory large B-cell lymphoma.
ALLO-501 (Allogene Therapeutics) is an allogeneic CAR-T derived from induced pluripotent stem cells from healthy donors that are genetically modified using transcription activator-like effector nucleases (TALEN) gene-editing technology.
The investigational cell therapy is being evaluated as part of the ALPHA2 study in combination with ALLO-647 (Allogene Therapeutics) — CD52-directed monoclonal antibody — for adults with relapsed or refractory large B-cell lymphoma.
The FDA placed a clinical hold on the ALPHA2 study in October 2021 after one patient with follicular lymphoma who received ALLO-501A showed evidence of a chromosomal abnormality.
The FDA lifted the hold in January after “investigations concluded that the chromosomal abnormality was unrelated to TALEN gene editing or Allogene’s manufacturing process,” according to a company-issued press release.
The most recent data from the study, presented at last year’s ASH Annual Meeting and Exposition, showed a 48% objective response rate among 25 patients who received ALLO-501A as consolidation therapy or as part of the trial’s dose-escalation phase.
Seven patients (28%) achieved complete response to therapy, with the longest ongoing complete response of more than 15 months.
Safety results showed neutropenia to be the most common treatment-related adverse event, occurring among 57% of patients. Researchers reported no cases of graft-versus-host disease.
All study patients experienced neurotoxicity and cytokine release syndrome, but investigators noted no cases of grade 3 or higher events during the study.
“The designation for ALLO-501A supports the patient need for access to an off-the-shelf CAR T product that can be delivered faster, more reliably and at greater scale,” Rafael Amado, MD, executive vice president of research and development and chief medical officer at Allogene, said in a company-issued press release.
“Patients who are eligible for autologous CAR T therapy are often faced with treatment delays and manufacturing failures, placing them at risk for disease progression and disease-related complications,” he added. “We look forward to initiating our pivotal trial on ALLO-501A and making this innovative product candidate readily available to patients.”
The FDA’s RMAT designation program — part of the 21st Century Cures Act — is designed to expedite review of regenerative medicine therapies intended to treat, modify, reverse or cure serious or life-threatening diseases or conditions. Preliminary clinical evidence must indicate the therapy has the potential to address unmet medical needs.
Reference:
Lekakis LJ, et al. Abstract 649. Presented at: ASH Annual Meeting and Exposition; Dec. 11-14, 2021; Atlanta.
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