TOKYO, Japan I April 24, 2017 I Daiichi Sankyo Company, Limited (hereafter, “Daiichi Sankyo”) announced today that the Duchenne muscular dystrophy (hereafter, “DMD”) treatment drug, DS-5141, being jointly developed with the Orphan Disease Treatment Institute Co., Ltd. (hereafter, “ODTI” *1) has been designated under the SAKIGAKE Designation System*2.
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.
*1 Orphan Disease Treatment Institute
Daiichi Sankyo jointly established the ODTI in 2013 with the Innovation Network Corporation of Japan and a fund managed by Mitsubishi UFJ Capital Co., Ltd.
*2 SAKIGAKE Designation System
SAKIGAKE Designation System is a core policy of the “Strategy of SAKIGAKE” (compiled by the Ministry of Health, Labour and Welfare in June, 2014) aimed at early introduction of innovative medicines, medical devices, etc. that are initially developed in Japan. The system’s objective is to designate drugs with prominent effectiveness against serious and life-threatening diseases in order to make them available to patients in Japan ahead of the rest of the world. Drugs are designated at a comparatively early stage of development and are given priority for clinical trial consultation and review.
SOURCE: Daiichi Sankyo
Post Views: 33
TOKYO, Japan I April 24, 2017 I Daiichi Sankyo Company, Limited (hereafter, “Daiichi Sankyo”) announced today that the Duchenne muscular dystrophy (hereafter, “DMD”) treatment drug, DS-5141, being jointly developed with the Orphan Disease Treatment Institute Co., Ltd. (hereafter, “ODTI” *1) has been designated under the SAKIGAKE Designation System*2.
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.
*1 Orphan Disease Treatment Institute
Daiichi Sankyo jointly established the ODTI in 2013 with the Innovation Network Corporation of Japan and a fund managed by Mitsubishi UFJ Capital Co., Ltd.
*2 SAKIGAKE Designation System
SAKIGAKE Designation System is a core policy of the “Strategy of SAKIGAKE” (compiled by the Ministry of Health, Labour and Welfare in June, 2014) aimed at early introduction of innovative medicines, medical devices, etc. that are initially developed in Japan. The system’s objective is to designate drugs with prominent effectiveness against serious and life-threatening diseases in order to make them available to patients in Japan ahead of the rest of the world. Drugs are designated at a comparatively early stage of development and are given priority for clinical trial consultation and review.
SOURCE: Daiichi Sankyo
Post Views: 33
