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Neurogene Announces RMAT Designation for NGN-401 Investigational Gene Therapy for Rett Syndrome
Designation based on preliminary clinical evidence from ongoing NGN-401 clinical trial that shows potential to address unmet medical needs in Rett syndrome
About this update from Neurogene Inc.
[{"type":"text","content":"\nDesignation based on preliminary clinical evidence from ongoing NGN-401 clinical trial that shows potential to address unmet medical needs in Rett syndrome\n\n\nRMAT designation provides an opportunity for an Accelerated Approval pathway under the 21st Century Cures Act, and is in addition to NGN-401’s selection by the FDA for the START Pilot Program\n\n\n NEW YORK--(BUSINESS WIRE)--\nNeurogene Inc. (NASDAQ: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that NGN-401 received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for the treatment of Rett syndrome. The designation was based on preliminary clinical evidence from the ongoing Phase 1/2 clinical trial with NGN-401 that shows potential to address unmet medical needs in this disease.\n\n\n“We appreciate the FDA’s ongoing commitment to expedite development of our NGN-401 gene therapy for Rett syndrome, with RMAT designation following NGN-401’s selection for the FDA’s START Pilot Program, two synergistic initiatives designed to more rapidly advance promising treatments for patients with unmet medical needs,” said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. “RMAT and START designations underscore the therapeutic potential of NGN-401 for Rett syndrome and reflect our commitment to accelerate development of NGN-401. We continue to expect to share interim efficacy data from the low-dose cohort in the fourth quarter of this year, with plans for additional data, including from the high-dose cohort, in the second half of 2025.”\n\n\nRMAT designation is granted for regenerative medicines intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, with preliminary clinical evidence that indicates that the drug has the potential to address unmet medical needs. Benefits of the RMAT designation program include all the benefits of Fast Track and Breakthrough Therapy designation programs, including early and frequent communications with FDA senior managers, intensive guidance on efficient drug development and eligibility for an Accelerated Approval pathway and Priority Review.\n\n\nIn addition, NGN-401 was previously selected by the FDA for its Support fo...