Clinical-stage biotechnology company ReviR Therapeutics announced on Wednesday that the US 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).

This 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.

RTX-117 is ReviR's investigational therapy developed through its 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 says that it is advancing the programme in CMT while expanding its translational research in ISR-related diseases, including fibrotic conditions.