RAG-18 granted FDA orphan drug status for Duchenne, Becker MD
Therapy being developed to counteract shortage of dystrophin in DMD, BMD
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The U.S. Food and Drug Administration (FDA) has granted orphan drug status to RAG-18, being developed as a potential treatment for both Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).
RAG-18 is a small activating RNA (saRNA) therapy from Ractigen Therapeutics that’s designed to counteract the shortage of dystrophin, the hallmark of both disorders.
Orphan drug designation is meant to encourage companies to develop therapies for rare conditions, defined as those affecting fewer than 200,000 people in the U.S., by providing a series of benefits, including seven years of market exclusivity upon approval and exemption from certain fees.
This announcement came about a month after the FDA granted the therapy rare pediatric disease designation for DMD. That status is similarly meant to encourage the development of therapies for rare diseases, but with a specific focus on those that mainly impact children.
“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,” Long-Cheng Li, MD, founder and CEO of Ractigen, said in a company press release.
“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,” Li added.
Orphan drug status follows rare pediatric disease designation for DMD
DMD and BMD are both caused by mutations in the DMD gene, which encodes a protein called dystrophin. In BMD, a type of muscular dystrophy in which the skeletal and heart muscles slowly degrade, this protein is generally present at low levels or functions incorrectly. In DMD, the most common form of the genetic disease, which leads to progressive deterioration of muscle fibers, dystrophin is usually missing altogether.
According to Ractigen, RAG-18 is a “first of its kind saRNA” that’s designed to activate the UTRN gene — which encodes a protein called utrophin — in muscle cells.
Utrophin is structurally and functionally similar to the dystrophin protein that is normally produced during embryonic development. Scientists believe that increasing the levels of utrophin during adulthood may compensate for the lack of dystrophin in DMD patients.
The experimental therapy is expected to induce the production of the protein and compensate for the lack of dystrophin in all DMD patients, regardless of the specific mutation location on the DMD gene.
Preclinical data showed that when delivered through an under-the-skin (subcutaneous) injection using the company’s proprietary lipid-conjugated oligonucleotide technology, RAG-18 was capable of mitigating muscle damage, supporting its therapeutic potential in DMD patients.
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