RARE Daily

FDA Grants Fast Track and Rare Pediatric Disease Designations to Wugen’s Cell Therapy for the Treatment of Rare Cancer

July 19, 2022

The U.S. Food and Drug Administration has granted Fast Track and Rare Pediatric Disease Designations for WU-CART-007, Wugen’s off-the-shelf CAR-T cell therapy, which is currently being explored for the treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL).

WU-CART-007 is an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR-T cell therapy engineered to overcome the technological challenges of harnessing CAR-T cells to treat CD7+ hematological malignancies. Wugen is deploying CRISPR/Cas9 gene editing technology to delete CD7 and the T-cell receptor alpha constant (TRAC), preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host-disease (GvHD). WU-CART-007 is manufactured using healthy donor-derived T-cells to eliminate the risk of malignant cell contamination historically observed in the autologous CAR-T setting. WU-CART-007 is currently being evaluated in a global phase 1/2 clinical trial for the treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL).

The FDA’s Fast Track is a process designed by the FDA to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. According to the FDA, the purpose is to get important new drugs to the patient earlier. Features of Fast Track designation include opportunities for more frequent interactions with the FDA review team and, if supported by clinical data, eligibility for Priority Review.

A rare pediatric disease designation is granted for drugs intended for the prevention or treatment of rare diseases that primarily affect individuals under 18 years old, with recipients of this designation potentially qualifying for a priority review voucher if certain conditions are met. The priority review voucher may be redeemed, transferred, or sold. Most recently, Novo Nordisk bought a priority review voucher from Marinus Pharmaceuticals for $110 million.

Author: Rare Daily Staff

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