FDA to Speed Up Review of Novel CAR-T Cell Therapy for Rare Forms of Lymphoma and Leukemia

A first-in-human trial is investigating the safety and efficacy of the novel CAR-T cell therapy in patients relapsed or refractory T-cell acute lymphoblastic leukemia/lymphoblastic lymphoma.

The Food and Drug Administration (FDA) granted a fast track designation to an investigational CAR-T cell therapy, WU-CART-007, for the treatment of relapsed or refractory T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL), according to the agent’s manufacturer, Wugen Inc.

Investigators are currently analyzing the safety and efficacy of WU-CART-007, an off-the-shelf CAR-T cell therapy, in a first-in-human clinical trial.

Oftentimes patients with blood cancers receive autologous CAR-T cell therapy. In this instance, a patient’s own immune cells are harvested and manufactured in a laboratory to better attack the cancer cells. However, some patients may have a low immune cell count, or their T cells are in poor shape.

Over recent years, more research has been focused on the development of allogeneic CAR-T cell treatments. These therapies, known as off-the-shelf treatments, utilize the immune cells from healthy donors. Those cells are then manufactured in a lab and may be stored for a period of time until a patient with cancer needs treatment with the off-the-shelf CAR-T cell therapy.

It is estimated that 44 patients aged 12 years and older will be enrolled onto the phase 1/2 trial.

The main aim of the study is to identify the occurrence of side effects from treatment, as well as the maximum tolerated dose and overall response rate (defined as the proportion of disease that fully or partially responds to treatment). The study authors also plan to analyze the duration of response (time of treatment response to time of disease relapse, progression or death from any cause) and progression-free survival (time from first treatment until disease progression).

The FDA also granted the novel CAR-T cell therapy a rare pediatric disease designation, which is given to treatments that focus on rare diseases that mainly affected children under the age of 18.

A fast track designation gives the agency the ability to interact frequently with a pharmaceutical company developing a drug. The goal with this process is expedite the review of drugs that fulfill an unmet medical need.

“Earlier this year, we dosed the first patient in our ongoing Phase 1/2 trial of WU-CART-007 for relapsed/refractory T-ALL/LBL and are currently in the dose escalation phase of the study,” Dan Kemp, president and CEO of Wugen, said in a news release. “We look forward to working closely with the FDA as we continue to advance WU-CART-007 through clinical development.”

The trial is expected to be complete by August 2026.

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