The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to NS-050/NCNP-03 for the treatment of Duchenne muscular dystrophy (DMD).
DMD is a rare disease affecting the muscles, leading to muscle wasting that gets worse over time. It is caused by genetic changes in the DMD gene, a gene responsible for encoding dystrophin, a protein involved in muscle cell structure and signaling pathways. DMD occurs primarily in males, though in rare cases may affect females. The most common symptom of DMD is progressive weakness and loss (atrophy) of both skeletal and heart muscle. Early signs of the disease may include delayed ability to sit, stand, or walk and difficulties learning to speak.
NS-050/NCNP-03 is an antisense oligonucleotide that produces a functional dystrophin protein with a shorter chain length. This targets the suppression of muscle function deterioration. A phase 1/2 clinical trial is being developed to test the safety and efficacy of NS-050/NCNP-03 in patients with DMD.
The FDA Rare Pediatric Disease Designation program incentives the development of novel drugs for rare pediatric diseases. Through the voucher program, companies receiving an approval for a drug may qualify for a voucher that gives priority review to the company for a different product. A Priority Review designation means the FDA must review the drug application within 6 months instead of the standard 10 months. The voucher can also be sold to another company and smaller companies often use that as an additional revenue source. Recently, Sarepta Therapeutics sold their voucher for $102 million.
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To learn more about DMD and other rare musculoskeletal diseases, visit https://checkrare.com/diseases/musculoskeletal-diseases/
