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ReviR Therapeutics Achieves Major Milestone with FDA Approval for RTX-117

ReviR Therapeutics, a pioneering biotech firm dedicated to innovating therapies for neurogenetic diseases, celebrated a significant breakthrough on October 13, 2025, when the U.S. Food and Drug Administration (FDA) awarded it Orphan Drug Designation (ODD) for RTX-117. This new therapy targets Charcot-Marie-Tooth disease (CMT), one of the most frequently inherited peripheral neuropathies, impacting between 125,000 and 150,000 individuals in the United States, and over 2.6 million worldwide.

CMT is known for progressive and debilitating symptoms, including muscle weakness and sensory loss, leading to considerable mobility challenges for many patients. Unfortunately, these individuals currently lack any approved treatment options, highlighting a critical medical need that ReviR aims to address with RTX-117.

Understanding RTX-117

RTX-117 represents a novel approach, designed to activate eIF2B, thereby normalizing cap-dependent mRNA translation and improving protein expression. This mechanism intends to tackle the root causes of CMT instead of merely alleviating its symptoms. With the potential to significantly slow or even halt disease progression, RTX-117 could revolutionize the management of this disorder and ultimately enhance neuromuscular function for patients.

ReviR is gearing up to initiate a Phase 1 clinical trial with healthy volunteers in early 2026, followed by an Ib/IIa study focusing specifically on CMT patients. This rigorous trial phase is critical to evaluate RTX-117's safety, tolerability, and pharmacokinetics, laying a solid foundation for its therapeutic promise.

The Importance of Orphan Drug Designation

The FDA’s Orphan Drug Designation program is integral in fostering the development of drugs intended for rare diseases, defined as those affecting less than 200,000 individuals in the U.S. This designation bestows various advantages including tax credits for clinical research, exemptions from specific FDA fees, and possibly seven years of market exclusivity after approval. For ReviR, achieving this status for RTX-117 is not only a validation of their scientific strategy but also a major boost that could expedite the clinical development timeline.

Paul August, PhD, Chief Scientific Officer at ReviR Therapeutics, emphasized the urgency of addressing CMT’s challenges. He stated, “Charcot-Marie-Tooth disease is one of the most prevalent inherited peripheral neuropathies worldwide, yet effective therapies that modify the course of the disease remain scarce. Receiving Orphan Drug Designation marks a critical step toward fulfilling this unmet medical need. Our preclinical research has highlighted RTX-117's ability to target the underlying pathophysiology of CMT, which is incredibly promising.”

Peng Yue, Chief Executive Officer of the company, added, “We see RTX-117 and our extensive pipeline as potential pathways to deliver impactful, disease-modifying therapies aimed at patients grappling with conditions that currently present limited or no treatment options.”

About ReviR Therapeutics

Founded in 2021, ReviR Therapeutics is on a mission to innovate therapies for serious neurogenetic disorders globally. Spearheaded by experts in computational biology and RNA research, the company’s focus is on developing effective, safe, and specific disease-modifying treatments. Aside from RTX-117, ReviR is exploring other oral small molecules aimed at RNA function modulation, indicating a strong commitment to expanding its portfolio in tackling significant medical challenges. For media inquiries or further information, visit ReviR Therapeutics.

In conclusion, ReviR Therapeutics’ receipt of Orphan Drug Designation for RTX-117 signifies a hope for many affected by Charcot-Marie-Tooth Disease. It represents a pivotal moment in the search for effective treatments and showcases the importance of innovative biotechnological advancements in medicine.