ReviR Therapeutics has received a rare paediatric disease designation (RPDD) from the US Food and Drug Administration (FDA) for RTX-117, its investigational oral small-molecule therapy for Charcot-Marie-Tooth disease (CMT).
The designation covers CMT as a whole and is not restricted to specific subtypes.
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The RPDD programme seeks to promote the development of therapies for rare, serious or life-threatening conditions that primarily affect children.
Under the programme, if RTX-117 receives approval and fulfils the statutory and programme criteria, ReviR Therapeutics may be eligible to obtain a rare paediatric disease priority review voucher.
This voucher can be used by the company or transferred to another sponsor for priority review of a future marketing application and may offer commercial value to ReviR Therapeutics.
CMT refers to a group of inherited neuromuscular disorders that often begin in childhood or adolescence.
Common symptoms include loss of sensation, muscle wasting, muscle weakness in the legs, pain, skeletal changes and slow motor development.
According to ReviR Therapeutics, there are currently no disease-modifying therapies approved for CMT.
RTX-117 has been developed using the company’s VoyageR AI platform and is intended to target the integrated stress response (ISR) pathway by activating eukaryotic initiation factor 2B (eIF2B), with the aim of restoring normal messenger ribonucleic acid (mRNA) translation and maintaining protein-expression balance.
The therapy is currently undergoing Phase I clinical trials, with completion expected by the end of the year. RTX-117 also holds orphan drug designation from the FDA for CMT and has received clinical trial authorisations in both China and the US.
ReviR Therapeutics chief scientific officer Paul August said: “People living with CMT, particularly children, urgently need more effective disease-modifying treatment options.
“This designation provides additional support for the continued development of RTX-117. As the CMT programme progresses, we will continue to build clinical and translational evidence that may inform our work in additional ISR-related diseases.”
ReviR Therapeutics focuses on developing therapies within neurology, oncology, immunology and inflammation.
The company aims to advance RTX-117 and other small-molecule candidates targeting RNA function across a range of ISR-related and fibrotic diseases.
