Rare Daily Staff
The U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy designation to CRISPR Therapeutics’ CTX110, its allogeneic CAR-T cell therapy targeting CD19+ B-cell malignancies.
CTX110, a wholly owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting Cluster of Differentiation 19, or CD19.
CTX110 is being investigated in the ongoing CARBON trial, a phase 1, single-arm, multi-center, open study designed to assess the safety and efficacy of several dose levels of CTX110 for the treatment of relapsed or refractory B-cell malignancies.
“This RMAT designation is based on the encouraging clinical data we have presented thus far, and it is an important milestone that recognizes the transformative potential of CTX110 for the treatment of hematological malignancies,” said Samarth Kulkarni, CEO of CRISPR Therapeutics. “We look forward to working closely with the FDA as we continue our efforts to bring this important new therapeutic modality to patients.”
Established under the 21st Century Cures Act, RMAT designation is a dedicated 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.
Similar to Breakthrough Therapy designation, 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.
Photo: Samarth Kulkarni, CEO of CRISPR Therapeutics