ReviR Therapeutics Receives Orphan Drug Designation from FDA for RTX-117 to Treat Charcot-Marie-Tooth Disease

BRISBANE, Calif. , Oct. 13, 2025 /PRNewswire/ -- ReviR Therapeutics, a biotech company focused on developing novel treatments for neurogenetic diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for RTX-117, its lead investigational therapy for Charcot-Marie-Tooth disease (CMT). ReviR is preparing to launch a Phase 1 clinical trial in healthy volunteers in early 2026 to evaluate the safety, tolerability, and pharmacokinetics of RTX-117, with a Phase Ib/IIa study in CMT patients planned to follow. 

CMT is the most prevalent inherited peripheral neuropathy, affecting an estimated 125,000 to 150,000 individuals in the United States and more than 2.6 million patients worldwide. It is a progressive, debilitating genetic disorder for which there are currently no approved treatment options, representing a significant unmet medical need.

RTX-117 is designed to activate eIF2B to restore translation of cap-dependent mRNAs to normalize protein expression, aiming to address an underlying cause of Charcot-Marie-Tooth disease rather than just alleviating symptoms. This differentiated approach has the potential to slow or halt disease progression and improve neuromuscular function. 

The FDA's Orphan Drug Designation program is a program designed to encourage the development for drugs and biologics that are intended for the treatment, prevention, or diagnosis of rare diseases or conditions affecting fewer than 200,000 people in the U.S. Benefits of the designation include tax credits for clinical research, exemption from certain FDA fees, and the potential for seven years of market exclusivity upon FDA approval.

"Charcot-Marie-Tooth disease represents one of the most prevalent inherited peripheral neuropathies in the world, yet effective disease-modifying therapies remain elusive," said Paul August, PhD, Chief Scientific Officer of ReviR Therapeutics, and a leading expert in neurodegenerative disease therapeutics. "The progressive nature of CMT is characterized by distal muscle weakness, sensory loss, and significant mobility impairment creating an urgent need for therapeutic intervention. Receiving Orphan Drug Designation for RTX-117 represents a pivotal milestone in addressing this critical unmet medical need. Our preclinical work has demonstrated RTX-117's potential to target the dysregulated pathophysiology of CMT. This FDA designation not only validates the scientific rationale behind our approach at ReviR, but also provides crucial regulatory support that will accelerate our clinical development timeline. We remain steadfast in our commitment to translating years of rigorous research into a therapeutic solution for the CMT patient community."

"We believe RTX-117 and our broader pipeline have the potential to deliver disease-modifying therapies to patients with serious conditions who currently have limited or no treatment options," added Peng Yue, Chief Executive Officer of ReviR Therapeutics.

About ReviR Therapeutics

ReviR Therapeutics is a global biotechnology company committed to discovering and developing innovative therapies for patients with serious neurogenetic diseases worldwide. ReviR was founded in 2021 by leaders in computational biology, RNA biology, and drug discovery, with the aim to create disease-modifying therapies that are highly specific, efficacious, and safe. ReviR's lead candidate, RTX-117, is an investigational small molecule therapy for Charcot-Marie-Tooth disease (CMT). In addition, ReviR's pipeline includes other orally administered small molecules designed to modulate RNA function. For more information about ReviR Therapeutics, visit www.revirtx.com or contact[email protected].

SOURCE ReviR Therapeutics