▎Reporting by the WuXi AppTec Content Team
On August 22, 2024, Ractigen Therapeutics announced that its small activating RNA (saRNA) drug RAG-18 received Orphan Drug Designation (ODD) from the U.S. FDA.Ractigen pointed out,FollowingLastMonthly RAG-18 AwardFDA Pediatric RareRare Pediatric Disease Designation (RPDD)The certification further establishes the potential of RAG-18 as an innovative treatment for Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD).Regardless of the type of DMD gene mutation, RAG-18 may become a promising therapeutic strategy with translational potential.。
Duchenne Muscular Dystrophy (DMD) is a severe X-linked recessive genetic disorder caused by mutations in the DMD gene, which encodes dystrophin.This disease primarily affects male children, with patients typically showing symptoms such as muscle inflammation, fibrosis, and reduced mobility for the first time between the ages of 3 to 5.If left untreated, most patients will lose the ability to walk independently before the age of 20 and may die before the age of 30 due to heart and diaphragm (respiratory muscle) failure.Data show that,The global incidence of DMD is approximately 1 in every 3,600 to 6,000 newborn males, with about 70 patients in China.000 people.RAG-18 is a first-in-class double-stranded saRNA drug that specifically targets and activates the expression of the UTRN gene in muscle cells through the RNA activation (RNAa) mechanism.Utrophin, encoded by the UTRN gene, is structurally and functionally similar to dystrophin. Its upregulation can functionally replace the missing dystrophin in DMD muscle cells, thereby treating all mutation types of DMD and BMD patients.Preclinical studies have shown that, using Ractigen's self-developed small nucleic acid delivery system LiCOTM and administered via subcutaneous injection, RAG-18 can significantly reduce muscle damage in DMD model mice, demonstrating potential for treating DMD patients.In July this year, RAG-18 received the Pediatric Rare Disease Drug Designation from the US FDA.Orphan Drug Designation Granted by FDA, aiming to encourage the development of drugs and biologics for the treatment of rare diseases.Companies that receive orphan drug designation will enjoy several development incentives, including seven years of market exclusivity from the date of market approval and a waiver of the new drug application fee.Dr. Longcheng Li, founder and CEO of Ractigen Therapeutics, stated: "Receiving the FDA’s Orphan Drug Designation is another key milestone achieved by RAG-18. Following the Pediatric Rare Disease Designation, this certification further affirms our pioneering work in the field of RNA activation and strengthens our resolve to bring transformative solutions to patients with rare diseases. This designation has injected us with renewed momentum as we continue to accelerate the development of RAG-18, committed to offering a revolutionary treatment option for DMD and BMD patients worldwide."”[1] RAG-18 Receives Dual FDA Orphan Drug and Pediatric Rare Disease Designations; Ractigen Accelerates Advancement in DMD Treatment. Retrieved Aug 22, 2024, from https://cn.ractigen.com/ractigen-therapeutics-announces-u-s-fda-orphan-drug-designation-odd-granted-to-rag-18-for-the-treatment-of-duchenne-muscular-dystrophy-and-becker-muscular-dystrophy/
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