Update of SAKIGAKE designation system in Japan – April, 2020 –

Twenty (20) pharmaceuticals have been designated as the accelerated review process of SAKIGAKE designation which started since 2015. 8 pharmaceuticals out of 20 products have been approved and 4 pharmaceuticals have been in phase III stages. Eleven (11) regenerative medicines have been designated as SAKIGAKE designation and 2 regenerative medicines have been approved. Two in vitro diagnostics have been designated as SAKIGAKE designation and the one has been approved.



Date Product Name Applicant Name Intended Indication Status
2016 Sirolimus(NPC-12G) Nobel Pharma Facial Angiofibroma(FA) caused by tuberous sclerosis complex(TSC) Approved[Mar/2018]

Price addition: 10%



2016 NS-065/NCNP-01


Nippon Shinyaku Duchenne muscular dystrophy:DMD Approved [MAR/2020]


2016 S-033188

Baloxavir marboxil

Shionogi Type A or B Influenza infection Approved[Feb/2018]

Price addition:10%


2016 BCX7353

selective inhibitor of plasma kallikrein

BioCryst Pharmaceuticals


the prevention of hereditary angioedema (HAE) attacks in patients diagnosed with HAE ODD[19NOV2019]
2016 ASP2215(Gilteritinib) Astellas Relapsed or Refractory Acute Myeloid Leukemia (AML) With FMS-like Tyrosine Kinase (FLT3) Mutation Approved [Sep 2018]

Price addition:10%


2016 Pembrolizumab MSD( Merck) gastric cancer which is impossible of curative resection Cancelled [2017/Sep]
2017 olipudase α Sanofi ニーマンピック病A/B型 – 酸性スフィンゴミエリナーゼ欠乏症 Phase II
2017 Aducanumab Biogen アルツハイマー病の進行抑制 Discontinuation
2017 DS-5141b Daiichi-Sankyo Duchenne muscular dystrophy PhaseI/II
2017 SPM-011



Stella Pharma Approved[MAR/2020]


2017 Nivolumab Ono Pharma biliary tract cancer Phase II
2018 RTA 402(バルドキソロンメチル)


Kyowa Hakko Kirin Diabetic kidney disease Phase III
2018 JR-141 JCR Pharmaceuticals Mucopolysaccharidosis II (Hunter syndrome) Phase III
2018 Tafamidis Meglumine Pfizer Transthyretin cardiac amyloidosis (TTR-CM) Approved[Mar 2019]


2018 tepotinib MSC2156119J Merck Serono Advanced (Stage IIIB/IV) non-small cell lung cancer harboring MET exon 14 skipping mutations ODD[19NOV2019]



2018 Trastuzumab deruxtecan


Daiichi Sankyo HER2-overexpressing unresectable advanced/ recurrent gastric cancer which have progressed after chemotherapy Phase II
2018 Entrectinib Ignyta/ Chugai Adult and pediatric patients with locally advanced or metastatic NTRK fusion-positive solid tumors, either whose disease progressed following prior therapy or who have no acceptable standard therapies Approved [JUN/2019]

Price addition:10%


2019 Valemetostat(DS3201) Daiichi-Sankyo 再発または難治性の末梢性T細胞性リンパ腫 Phase II
2019 イキサゾミブクエン酸エステル

ixazomib citrate

Takeda ALアミロイドーシス Discontinuation


2019 TAK-925 Takeda Narcolepsy Phase I
2019 ASP-1929 Rakuten Medical 頭頸部癌/Head and Neck Cancer Phase III
2019 E7090 Esai biliary tract cancer Phase II

Regenerative Medicine

2016 STR01


Nipro 骨髄損傷


Approved [Dec/2018]


2016 G47A Daiichi-Sankyo 悪性脳腫瘍(神経膠腫)


Phase II
2016 JRM-001


日本再生医療 小児先天性心疾患


Phase III
2017 CLS2702C/D


Cellseed 食道がん手術後


Phase III
2017 非自己iPS細胞由来ドパミン神経前駆細胞 DaiNipponSumito(DNP) パーキンソン病


Phase I/II
2017 ヒト(同種)成人骨髄由来多能性前駆細胞 Hellios 急性期の脳梗塞 Phase II/III
2018 TBI-1301



Takara Bio


The gene of the receptor that specifically recognizes the tumor antigen is transduced ex-vivo to lymphocytes of patients, and the gene-modified lymphocytes are infused back to the patients. It acquires the capability to attack cancer cells and the treatment of synovial sarcoma. PhaseI/II
2018 CLBS12


Caladrius Biosciences By isolating CD34 + cells from the patients’ own peripheral blood and infuse back to the patients, CD34 + cells promote angiogenesis, resulting in an improvement from critical limb ischemia conditions. PhaseII
2018 AVXS-101




This product is for the treatment of spinal muscular atrophy (SMA), by transducing SMN gene into patients, producing SMN protein in the body and supplementing, then improving the functions of nerve and skeletal muscle. Approved [MAR/2020]
2019 OBP-301(テロメライシン) Oncolys / Chugai 食道癌(Esophageal cancer) PhaseII
2019 SB623 SunBio 外傷性脳損傷における運動障害改善 Phase II

In vitro diagnostics

2016 Titanium Bridge Nobel Pharma Launched [2018/06/01]
2016 Otsuka Cancelled


人工気管 DaiichiIka
2017 BNCTシステム Sumitomojuki ホウ素中性子補足療法
2017 UT-Heart UT-Heart 心臓シュミレーションプログラム
2017 がん関連遺伝子パネル検査システム シスメックス Approved [Dec/2018]
2018 Cardiovascular-patch

OFT-G1 (tentative)

Teijin Cardiovascular-patch consisting of absorbable synthetic polymer stitches, non-absorbable synthetic polymer stitches and cross-linked gelatin membrane, used for correction of vessels, securing of blood flow, construction /reconstruction of surrounding tissues during cardiovascular surgery.
2018 CliniMACS

CD34 System


Miltenyi Biotec Selectively isolate CD34 + cells, and administer obtained CD34 + cell with collagen soft tissue injection (scaffold material) to the refractory bone fracture (nonunion) site, and accelerate bone adhesion.
2019 マイクロ波マンモグラフィ Integral Geometry Science
2019 下肢動脈バイパス用人工血管作製用鋳型 バイオチューブ


2019 リン酸化プルランバイオアドヒーシブ BioARC
2019 DNAチップによる膵臓・胆道癌検査キット 東レ