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news.The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Ractigen Therapeutics' RAG-18, a small activating RNA (saRNA) product candidate for the treatment of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).
The saRNA product candidate is being developed for the treatment of Duchenne muscular dystrophy and Becker muscular dystrophy. Credit: Michal Jarmoluk from Pixabay.
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RAG-18 is claimed to be the first saRNA therapeutic to receive ODD status.
DMD and BMD are caused by mutations in the DMD gene.
The FDA grants ODD to drugs targeting conditions affecting fewer than 200,000 individuals in the US, providing incentives such as seven-year marketing exclusivity upon market approval and a new drug application (NDA) fee waiver.
The status follows the rare pediatric disease designation (RPDD) awarded to RAG-18 last month for addressing rare muscular dystrophies.
RAG-18 is designed to activate UTRN gene expression in muscle cells through an RNA activation (RNAa) mechanism.
The utrophin protein, encoded by the UTRN gene, shares similarities with dystrophin and could act as a functional substitute in DMD muscle cells.
This approach aims to offer a universal treatment for DMD patients, irrespective of their specific genetic mutation.
Preclinical studies have shown that the asset administered via subcutaneous injection with Ractigen’s lipid-conjugated oligonucleotide (LiCO) technology, can significantly reduce muscle damage, indicating a promising treatment avenue for DMD.
Ractigen Therapeutics founder and CEO Dr Long-Cheng Li said: “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 RNAa 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.”
