RARE Daily

FDA Grants CRISPR Therapeutics RMAT Designation for Cutaneous T Cell Lymphomas

September 29, 2022

The U.S. Food and Drug Administration granted CRISPR Therapeutics Regenerative Medicine Advanced Therapy (RMAT) designation for its CTX130, the company’s allogeneic CAR T cell therapy targeting CD70, for the treatment of Mycosis Fungoides and Sézary Syndrome, two forms of cutaneous T cell lymphoma.

Photo: Phuong Khanh Morrow, chief medical officer of CRISPR Therapeutics

CTX130 is a healthy, donor-derived, gene-edited, allogeneic CAR T experimental therapy targeting Cluster of Differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies.

CTX130 is being investigated in two ongoing independent phase 1 single-arm, multi-center, open-label clinical trials that are designed to assess the safety and efficacy of several dose levels of CTX130 in adult patients. The COBALT-LYM trial is evaluating the safety and efficacy of CTX130 for the treatment of relapsed or refractory T or B cell malignancies. The COBALT-RCC trial is evaluating the safety and efficacy of CTX130 for the treatment of relapsed or refractory renal cell carcinoma. CTX130 has received Orphan Drug and Regenerative Medicine Advanced Therapy designations from the FDA.

“The RMAT designation is an important milestone for the CTX130 program that recognizes the transformative potential of our cell therapy in patients with T-cell lymphomas based upon encouraging clinical data to date,” said Phuong Khanh Morrow, chief medical officer of CRISPR Therapeutics.

RMAT designation is a program designed to expedite the drug development and review processes for promising pipeline products, including genetic therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition.

RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application, and other opportunities to expedite development and review.

Author: Rare Daily Staff

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