Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to RAG-18, one of the company’s lead saRNA product candidates. This designation follows the recent Rare Pediatric Disease Designation (RPDD) granted to RAG-18 last month, further solidifying the potential of this novel therapeutic strategy for Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) caused by any mutation of the DMD gene. These milestones establish RAG-18 as the world’s first saRNA drug to achieve such significant regulatory recognition from the FDA.
Orphan drug designation is granted by the FDA to a drug or biologic intended to treat a rare disease or condition, which generally includes a disease or condition that affects fewer than 200,000 individuals in the U.S. The designation provides companies with development incentives, including a seven-year marketing exclusivity from the date of market approval and a waiver of the New Drug Application fee.
Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics, stated: “Receiving FDA Orphan Drug Designation marks a pivotal achievement for RAG-18. Combined with the recent Rare Pediatric Disease Designation, it reflects the groundbreaking work we’re doing with RNA activation and reinforces our commitment to making a real difference in the lives of those affected by rare diseases. This recognition fuels our determination to push forward with RAG-18’s development, aiming to bring innovative and life-changing treatments to DMD and BMD patients around the world.”
