| Generic Name |
Date Designated |
Orphan Designation |
Sponsor Company |
| (R)-2-methyl-6-nitro-2-{4-[4-(4-trifluoromethoxyphenoxy)piperidin-1-yl]phenoxymethyl}-2,3-dihydroimidazo[2,1-b]oxazole |
07/12/2007 |
Treatment of pulmonary tuberculosis. |
Otsuka Pharmaceutical Company, Ltd |
| 5-{[(3R,4R)-1-(4-chloro-2,6-difluorophenyl)-3,4-dihydroxypiperidin-4-yl]methoxy}-8-fluoro-3,4-dihydroquinolin-2(1H)-one |
12/05/2016 |
Treatment of active tuberculosis |
Otsuka Pharmaceutical Company, Ltd |
| 7-ethyl-10-hydroxycamptothecin (EHC) |
04/13/2016 |
Treatment of small cell lung cancer. |
Nippon Kayaku Co., Ltd. |
| Busulfan |
07/28/1994 |
As preparative therapy in the treatment of malignancies with bone marrow transplantation. |
Otsuka Pharmaceutical Company, Ltd |
| camostat |
05/18/2011 |
Treatment of chronic pancreatitis |
NIXS Corporation |
| Cis-diamminedichloroplatinum (II) polyethylene glycol-polyglutamate (cis platinum PEG-pGlu) polymeric micelles |
07/27/2017 |
Treatment of biliary tract carcinoma |
NanoCarrier Co., Ltd |
| Darinaparsin |
09/13/2010 |
Treatment of peripheral T-cell lymphoma. |
Solasia Pharma K.K. |
| Dendritic cell/tumor cell fusion vaccine + recombinant human interleukin 12 |
01/25/2017 |
Treatment of malignant glioma |
CyTIX Inc. |
| Dextran sulfate sodium |
11/19/1987 |
Treatment of aquired immunodeficiency syndrome. |
Ueno Fine Chemicals Industry, Ltd. |
| edaravone |
05/12/2015 |
Treatment of amyotrophic lateral sclerosis |
Mitsubishi Tanabe Pharma Corporation |
| epirubicin-conjugated polymer micelles |
07/13/2017 |
Treatment of soft tissue sarcoma |
NanoCarrier Co., Ltd |
| H-(D)p-Benzoylphenylalanyl-(D)seryl-(D)tryptophanyl-(D)seryl-(D)pentafluorophenylalanyl-(D)cyclohexylalanyl-(D)arginyl-(D)arginyl-(D)arginyl-(D)glutaminyl-(D)arginyl-(D)arginine acetate salt |
12/22/2011 |
For use in combination with cisplatin and pemetrexed for the treatment of patients with mesothelioma |
CanBas Company, Ltd. |
| Humanized IgG-1 antibody targeting human transferrin receptor conjugated to human iduronate-2-sulfatase |
10/15/2018 |
Treatment of Mucopolysaccharidosis Type II (MPS II) or Hunter Syndrome |
JCR Pharmaceuticals Co., Ltd. |
| N-{2-[4-(2-Amino-5-chloropyridin-3-yl)phenoxy]pyrimidin-5-yl}-N’-[2-(methanesulfonyl)-5-(trifluoromethyl)phenyl]urea mono-4-toluenesulfonate |
09/13/2017 |
Treatment of tropomyosin receptor kinase (Trk) gene fusion-positive cancer |
Ono Pharmaceuticals Co. Ltd. |
| polyphenon E |
07/17/2008 |
Treatment of chronic lymphocytic leukemia |
Mitsui Norin Co., Ltd |
| Prostaglandin E1 enol ester (AS-013) |
06/12/1998 |
Treatment of Fontaine Stage IV chronic critical limb ischemia. |
LTT Baio-Pharma Co., Ltd |
| sirolimus |
05/17/2017 |
Treatment of angiofibroma associated with tuberous sclerosis |
NobelPharma Co., LTD |
| small interfering RNA agent that targets human transforming growth factor-ß1 messenger RNA |
12/19/2018 |
Treatment of Idiopathic pulmonary fibrosis |
Bonac Corporation |
| Telomerase Specific Replication-competent Oncolytic Adenovirus |
06/01/2020 |
Treatment of esophageal cancer |
Oncolys BioPharma Inc. |
| tolvaptan |
04/06/2012 |
Treatment of autosomal dominant polycystic kidney disease |
Otsuka Pharmaceutical Company, Ltd. |
| unoprostone isopropyl |
09/16/2010 |
Treatment of retinitis pigmentosa |
R-Tech Ueno, Ltd. |
| Yttrium(90Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10 |
12/03/2012 |
Treatment of soft tissue sarcoma |
OncoTherapy Science, Inc. |
オーファンドラッグ指定は、薬事申請認可ではありませんが、FDAが将来性・可能性を審査して有望なものにしか指定を与えません。ですから、日本のがん免疫治療で、これを得るということがどれだけ大変なことだか、ご想像頂ければ幸いです。
今日まで、日本企業でこの指定を受けたものは２２件しかありません。全リストが下記のとおりです。
今後は、販売認可を目指して研究開発を続けていく予定です