Daiichi Sankyo has announced that the Duchenne muscular dystrophy (DMD) treatment drug, DS-5141, being jointly developed with the Orphan Disease Treatment Institute Co., Ltd. (ODTI) has been designated under the SAKIGAKE Designation System.

DMD is known to be a disease that affects one in 3,500 new-born males regardless of ethnicity. DMD occurs because muscle cells of DMD patient do not produce dystrophin. However, current treatments are extremely limited, and their effects are also limited.

Because DS-5141b induces exon 45 skipping of a dystrophin mRNA to promote incomplete but functional dystrophin production, it is expected to be an effective treatment for DMD. In addition, DS-5141 contains the active ingredient ENA oligonucleotide, a modified nucleic acid made using proprietary technology owned by Daiichi Sankyo. A phase 1/2 clinical trial of the drug is currently underway in Japan.