ReviR Therapeutics获得FDA罕见儿科疾病认定（RPDD），其候选药物RTX-117用于腓骨肌萎缩症（CMT）

ReviR Therapeutics Receives FDA Rare Pediatric Disease Designation for RTX-117 in Charcot-Marie-Tooth Disease
Designation supports the global development of RTX-117 and may provide potential future access to a transferable Priority Review Voucher, subject to approval and applicable requirements
BRISBANE, Calif., Aug. 26, 2026 /PRNewswire/ -- ReviR Therapeutics, a clinical-stage biotechnology company advancing small-molecule therapies through the integration of artificial intelligence and RNA biology, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to RTX-117, the company's investigational oral small-molecule candidate, for its development in Charcot-Marie-Tooth disease (CMT). The designation applies to CMT broadly and is not limited to a specific subtype.
The FDA's RPDD program is intended to encourage the development of therapies for serious or life-threatening rare diseases that predominantly affect children. If RTX-117 is ultimately approved and meets applicable statutory requirements and program criteria, ReviR may be eligible to receive a Rare Pediatric Disease Priority Review Voucher (PRV). The voucher may be transferred to another sponsor or redeemed to obtain priority review for a future marketing application and may hold potential commercial value for ReviR.
CMT comprises a group of progressive inherited neuromuscular disorders that often emerge during childhood or adolescence. Patients may experience leg weakness and muscle wasting, skeletal deformities, pain, sensory impairment and delayed motor development. Despite the longstanding need for new treatment options, there are currently no approved disease-modifying therapies for CMT.
RTX-117 is ReviR's investigational therapy developed through ReviR's proprietary VoyageR AI platform and is designed to modulate the integrated stress response (ISR) pathway. By activating eIF2B, RTX-117 is intended to restore normal mRNA translation and protein-expression homeostasis. ReviR is advancing the program in CMT while expanding its translational research in ISR-related diseases, including fibrotic conditions.
Previously, RTX-117 has received FDA Orphan Drug Designation(ODD) for CMT and clinical trial authorizations in both the United States and China. The program is currently in Phase 1 clinical development, with completion of the Phase 1 study anticipated by the end of this year.
"People living with CMT, particularly children, urgently need more effective disease-modifying treatment options," said Paul R. August, Ph.D., Chief Scientific Officer of ReviR Therapeutics. "This designation provides additional support for the continued development of RTX-117. As the CMT program progresses, we will continue to build clinical and translational evidence that may inform our work in additional ISR-related diseases."
"RPDD adds another important strategic dimension to the global development of RTX-117," said Peng Yue, Ph.D., Co-founder and CEO of ReviR Therapeutics. "The experience and capabilities we build through RTX-117 will help inform the expansion of our small-molecule RNA platform into a broader range of serious diseases, including genetic diseases, immunology and inflammation, and oncology. We look forward to working with partners worldwide to advance new therapeutic options for patients."
About ReviR Therapeutics
ReviR Therapeutics is a clinical-stage global biotechnology company developing innovative therapies for patients with serious and difficult-to-treat diseases across neurology, immunology and inflammation, and oncology. Founded in 2021 by leaders in computational biology, RNA biology, genomics and drug discovery, ReviR integrates artificial intelligence with RNA biology to create disease-modifying therapies designed to be highly specific, effective and safe. ReviR's lead clinical-stage program, RTX-117, is advancing across multiple ISR-related disease areas, including CMT and VWM, with fibrotic diseases among the additional indications under exploration. ReviR's broader pipeline includes orally administered small molecules designed to modulate RNA function by splicing. For more information, visit www.revirtx.com.
About RTX-117
RTX-117 is a potential first-in-class, orally administered small-molecule therapy developed by ReviR Therapeutics, with discovery and molecular design supported by XtalPi's AI and robotics-enabled drug discovery platform. RTX-117 is designed to modulate the integrated stress response (ISR) pathway through activation of eIF2B, a key regulator of protein translation. By restoring mRNA translation and protein-expression homeostasis, the program aims to address molecular mechanisms implicated in ISR-related diseases.
RTX-117 is currently in a Phase 1 clinical trial, with Investigational New Drug (IND) clearance secured for CMT and VWM in the United States and China. In parallel, ReviR is expanding development of RTX-117 into additional ISR-related indications, including fibrotic diseases.
Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding the clinical development, potential therapeutic applications, regulatory pathway and future development of RTX-117, as well as ReviR's potential eligibility for a Rare Pediatric Disease Priority Review Voucher. These statements are based on current expectations and are subject to risks and uncertainties that could cause actual results to differ materially, including risks related to clinical development, preclinical findings, regulatory review, program timing and other factors. Preclinical findings may not be replicated in clinical trials, and RTX-117 has not been approved by the FDA or any other regulatory authority. ReviR undertakes no obligation to update these forward-looking statements except as required by applicable law.
SOURCE ReviR Therapeutics