ReviR Therapeutics Receives FDA Orphan Drug Designation for CMT Therapy RTX-117

BRISBANE, Calif. – October 13, 2025 – ReviR Therapeutics, a biotechnology company developing 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).

CMT is the most prevalent inherited peripheral neuropathy, affecting an estimated 125,000 to 150,000 individuals in the United States. It is a progressive, debilitating genetic disorder with no currently approved treatment options, representing a significant unmet medical need.

Targeting the Underlying Cause of CMT
RTX-117 employs a differentiated approach designed to target an underlying cause of CMT rather than just alleviating symptoms. The therapy is designed to activate eIF2B to restore the translation of cap-dependent mRNAs, thereby normalizing protein expression. This mechanism has the potential to slow or halt disease progression and improve neuromuscular function.

The FDA’s ODD program provides incentives for developing drugs and biologics that treat rare diseases, including tax credits and the potential for seven years of market exclusivity upon approval.

“Receiving Orphan Drug Designation for RTX-117 represents a pivotal milestone in addressing this critical unmet medical need,” said Paul August, PhD, Chief Scientific Officer of ReviR Therapeutics. “Our preclinical work has demonstrated RTX-117’s potential to target the dysregulated pathophysiology of CMT.”

Clinical Trials Set to Launch in 2026
ReviR is preparing to launch a Phase 1 clinical trial in healthy volunteers in early 2026 to evaluate the therapy’s safety, tolerability, and pharmacokinetics. A subsequent Phase Ib/IIa study in CMT patients is planned to follow. The company’s approach is focused on delivering disease-modifying therapies to patients with serious conditions who currently have limited or no treatment options.