This small interfering ribonucleic acid (RNA)[siRNA] therapy is designed to combat one of the ALS subtypes, FUS-ALS, by reducing FUS protein levels.
RAG-21 leverages RNA interference (RNAi) technology to selectively reduce FUS messenger RNA (mRNA) transcripts, eliminating the development of toxic proteins that contribute to the disease’s progression.
Delivered through the SCAD platform, RAG-21 claims to have shown promise in preclinical studies, demonstrating potent efficacy and safety in mitigating FUS cytoplasmic mis-localisation and aggregation.
This FDA designation is the second for the company, following RAG-17’s orphan drug status for superoxide dismutase 1 (SOD1)-ALS.
The ODD offers several benefits, including a 25% tax credit on qualified clinical trial expenditures, seven years of marketing exclusivity post-approval, and an exemption from the new drug application (NDA) fee.
A debilitating neurodegenerative condition, ALS severely affects patients’ quality of life, often leading to respiratory failure within two to five years of diagnosis.
Ractigen Therapeutics founder and CEO Dr Long-Cheng Li said: “The FDA’s Orphan Drug Designation for RAG-21 underscores the critical need for innovative therapies targeting ALS, particularly for patients with FUS mutations.
“FUS-ALS represents one of the most severe and rapidly progressing subtypes of ALS, with no curative treatments currently available. We are committed to developing innovative therapies like RAG-21 to provide meaningful treatment options for patients with ALS and other life-threatening rare diseases.”
In August this year, the company received ODD status from the FDA for RAG-18 to treat Becker muscular dystrophy and Duchenne muscular dystrophy.