＜Japan＞
August 07,2025
| Stage | Code No. (Generic name) |
Therapeutic field | Indications | Origin | Development |
|---|---|---|---|---|---|
| Launch Phase III |
NS-065/NCNP-01 (viltolarsen) |
intractable and rare diseases | Duchenne muscular dystrophy | Co-development with National Center of Neurology and Psychiatry |
Nippon Shinyaku Co., Ltd. |
| Filed | NS-401 (tagraxofusp) |
hematologic malignancies | blastic plasmacytoid dendritic cell neoplasm | In-licensed from The Menarini Group |
Nippon Shinyaku Co., Ltd. |
| Phase III | ZX008 (fenfluramine hydrochloride) |
intractable and rare diseases | CDKL5 deficiency disorder | Distribution partnership with UCB S.A. (formerly known as Zogenix, Inc.) |
UCB S.A. (formerly known as Zogenix, Inc.) |
| Phase III | GA101 (obinutuzumab) |
intractable and rare diseases | lupus nephritis | In-licensed from Chugai Pharmaceutical Co., Ltd. |
Co-development with Chugai Pharmaceutical Co., Ltd. |
| Phase III | GA101 (obinutuzumab) |
intractable and rare diseases | pediatric nephrotic syndrome | In-licensed from Chugai Pharmaceutical Co., Ltd. |
Co-development with Chugai Pharmaceutical Co., Ltd. |
| Phase III | GA101 (obinutuzumab) |
intractable and rare diseases | extra renal lupus | In-licensed from Chugai Pharmaceutical Co., Ltd. |
Co-development with Chugai Pharmaceutical Co., Ltd. |
| Phase III | LY3527727 (pirtobrutinib) |
hematologic malignancies | mantle cell lymphoma | Alliance agreement in Japan with Eli Lilly Japan K.K. |
Eli Lilly Japan K.K. |
| Phase III | LY3527727 (pirtobrutinib) |
hematologic malignancies | chronic lymphocytic leukemia | Alliance agreement in Japan with Eli Lilly Japan K.K. |
Eli Lilly Japan K.K. |
| Phase II | NS-304 (selexipag) |
cardiovascular | arteriosclerosis obliterans | Nippon Shinyaku Co., Ltd. | Nippon Shinyaku Co., Ltd. |
| Phase II | NS-580 | gynecology | endometriosis | Nippon Shinyaku Co., Ltd. | Nippon Shinyaku Co., Ltd. |
| Phase II | NS-580 | urological diseases | chronic prostatitis / chronic pelvic pain syndrome | Nippon Shinyaku Co., Ltd. | Nippon Shinyaku Co., Ltd. |
| Phase II | NS-089/NCNP-02 (brogidirsen) |
intractable and rare diseases | Duchenne muscular dystrophy | Co-development with National Center of Neurology and Psychiatry |
Nippon Shinyaku Co., Ltd. |
| Phase II | NS-229 | intractable and rare diseases | eosinophilic granulomatosis with polyangiitis | Nippon Shinyaku Co., Ltd. | Nippon Shinyaku Co., Ltd. |
| Phase I/II | NS-050/NCNP-03 | intractable and rare diseases | Duchenne muscular dystrophy | Co-development with National Center of Neurology and Psychiatry |
Nippon Shinyaku Co., Ltd. |
| Phase I | NS-917 (radgocitabine) |
hematologic malignancies | relapsed/refractory acute myeloid leukemia | In-licensed from Delta-Fly Pharma, Inc. |
Nippon Shinyaku Co., Ltd. |
| Phase I | NS-025 | urological diseases | urological diseases | Nippon Shinyaku Co., Ltd. | Nippon Shinyaku Co., Ltd. |
| Phase I | NS-863 | cardiovascular | cardiovascular diseases | Nippon Shinyaku Co., Ltd. | Nippon Shinyaku Co., Ltd. |
＜Overseas＞
| Stage | Code No. (Generic name) |
Therapeutic field | Indications | Origin | Development |
|---|---|---|---|---|---|
| U.S. Launch Phase III |
NS-065/NCNP-01 (viltolarsen) |
intractable and rare diseases | Duchenne muscular dystrophy | Co-development with National Center of Neurology and Psychiatry |
Nippon Shinyaku Co., Ltd. |
| Filed | CAP-1002 (deramiocel) |
intractable and rare diseases | Duchenne muscular dystrophy cardiomyopathy | Partnered with Capricor Therapeutics, Inc. |
Capricor Therapeutics, Inc. |
| Filed | RGX-121 (clemidsogene lanparvovec) |
intractable and rare diseases | Mucopolysaccharidosis Type II | Partnered with REGENXBIO Inc. |
REGENXBIO Inc. |
| Phase III | CAP-1002 (deramiocel) |
intractable and rare diseases | Duchenne muscular dystrophy | Partnered with Capricor Therapeutics, Inc. |
Capricor Therapeutics, Inc. |
| Phase II | NS-089/NCNP-02 (brogidirsen) |
intractable and rare diseases | Duchenne muscular dystrophy | Co-development with National Center of Neurology and Psychiatry |
Nippon Shinyaku Co., Ltd. |
| Phase II | NS-229 | intractable and rare diseases | eosinophilic granulomatosis with polyangiitis | Nippon Shinyaku Co., Ltd. | Nippon Shinyaku Co., Ltd. |
| Phase I/II | NS-050/NCNP-03 | intractable and rare diseases | Duchenne muscular dystrophy | Co-development with National Center of Neurology and Psychiatry |
Nippon Shinyaku Co., Ltd. |
| Phase I/II | ATSN-101 | intractable and rare diseases | GUCY2D-associated Leber congenital amaurosis | In-licensed from Atsena Therapeutics, Inc. |
Atsena Therapeutics, Inc. |
| Phase I/II | RGX-111 | intractable and rare diseases | Mucopolysaccharidosis Type I | Partnered with REGENXBIO Inc. |
REGENXBIO Inc. |