Allogene FDA Grants RMAT Designation to Allogene’s ALLO-501A for Large B Cell Lymphoma

Rare Daily Staff

The U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy (RMAT) designation to Allogene’s experimental allogeneic CAR T therapeutic ALLO-501A in the rare cancer relapsed/refractory large B cell lymphoma.

“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,” said Rafael Amado, executive vice president of Research and Development and chief medical officer of Allogene. “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. We look forward to initiating our pivotal trial on ALLO-501A and making this innovative product candidate readily available to patients.”

The RMAT designation followed positive data from the ALPHA2 trial and was based on the potential of ALLO-501A to address the unmet need for patients who have failed other therapies, which was presented at the American Society of Hematology (ASH) 2021 Annual Meeting. The data support the potential of ALLO-501A to provide a safe and durable alternative to approved autologous CAR T therapies in CAR T naïve patients.

ALLO-501A was associated with consistent and manageable safety with no dose limiting toxicities (DLTs) or graft-vs-host disease (GvHD) and minimal Grade 3 Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), or Grade 3 cytokine release syndrome (CRS).

There were no relapses observed in LBCL CAR T naïve patients who were in a complete response (CR) at six months with the longest ongoing CRs with ALLO-501A at 15+ months.

Nearly all enrolled patients were able to receive therapy with the median time from enrollment to initiation of treatment of two days in ALPHA2.

Allogene intends to initiate a phase 2 pivotal trial of ALLO-501A in mid-2022.

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the development and review processes for promising pipeline products, including cell therapies, that includes all the benefits of Fast Track and Breakthrough designation. An investigational cell therapy is eligible for RMAT designation if it is intended to treat, modify, reverse, or cure a serious or life-threatening disease; and preliminary clinical evidence indicates that the therapy has the potential to address unmet medical needs for that disease. Advantages of the RMAT designation include early interactions with FDA that may be used to discuss potential surrogate or intermediate endpoints and potential ways to satisfy post approval requirements.

Photo: Rafael Amado, executive vice president, head of R&D, and chief medical officer at Allogene