FDA Grants Rare Pediatric Disease Designation to ReviR’s RTX-117

The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to ReviR Therapeutics’ investigational drug RTX-117 for the treatment of Charcot-Marie-Tooth disease (CMT).

The designation applies broadly to CMT and is not limited to a specific type of the disease. RTX-117 is an oral small-molecule therapy currently being developed in Phase 1 clinical trials.

Charcot-Marie-Tooth disease is a group of inherited disorders that affect the nerves and muscles. Symptoms often begin during childhood or adolescence and can include leg weakness, muscle loss, pain, problems with sensation, skeletal changes and delayed motor development.

There are currently no approved disease-modifying treatments for CMT, creating a significant need for new therapies that could address the underlying causes of the disease.

RTX-117 was developed using ReviR Therapeutics’ VoyageR artificial intelligence platform and is designed to target the integrated stress response (ISR) pathway. The drug works by activating eIF2B, with the aim of restoring normal messenger RNA translation and maintaining healthy protein production in cells.

ReviR is developing RTX-117 as a potential treatment for CMT while also studying the ISR pathway in other diseases, including fibrotic conditions.

The company previously received FDA Orphan Drug Designation for RTX-117 in CMT. The program has also received clinical trial authorizations in the United States and China.

The Phase 1 clinical study of RTX-117 is currently underway, with ReviR expecting to complete the study by the end of 2026. Data from the study will help the company assess the drug’s safety and support future clinical development.

The FDA’s Rare Pediatric Disease program is designed to encourage the development of treatments for serious or life-threatening diseases that mainly affect children. If RTX-117 is eventually approved and meets the program’s requirements, ReviR could become eligible for a Rare Pediatric Disease Priority Review Voucher.

Such a voucher can potentially be transferred to another company or used to receive priority review for a future marketing application, giving it potential commercial value.

ReviR said the latest FDA designation provides additional support for the development of RTX-117 and could also help guide the company’s broader research into ISR-related diseases.

The company plans to continue developing its RNA-focused small-molecule platform across areas including genetic diseases, immunology and inflammation, and oncology.