Elixirgen Therapeutics, Inc., a clinical-stage biotechnology company focused on treating rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to EXG-34217, a gene therapy for treatment of Telomere Biology Disorders (TBDs). EXG-34217 is currently being studied in an ongoing Phase 1/2 trial (NCT04211714) for the treatment of patients with TBDs with bone marrow failure. Enrollment is currently open for participants aged 12 and older, regardless of gender or ethnicity.
RMAT designation, established under the 21st Century Cures Act, is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including gene and cell 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 therapy has the potential to address unmet medical needs for that disease. A treatment with RMAT designation receives all the benefits of Fast Track and Breakthrough Therapy designation programs, including early interactions with the FDA, which may be used to discuss potential surrogate or intermediate endpoints to support accelerated approval.
