中國創(chuàng)新藥企業(yè)2023年獲得FDA孤兒藥資格盤點

熱門推薦： FDA , 創(chuàng)新藥 , 孤兒藥 ,

作者：zhulikou431 來源：zhulikou431

2024-01-22

近年來，國內(nèi)多家生物醫(yī)藥公司開發(fā)的創(chuàng)新藥在美國獲得孤兒藥資格認(rèn)定，本文我們就盤點一下2023年中國制藥企業(yè)獲得FDA孤兒藥資格認(rèn)定的創(chuàng)新藥。

2024年1月8日，F(xiàn)DA的CDER發(fā)布了《2023年新藥治療審批報告》，從這個審批報告中我們可以了解到，2023年FDA批準(zhǔn)了55種創(chuàng)新藥物，這個數(shù)量遠(yuǎn)超于2022年的37種。另外，值得我們注意的是這55款獲批新藥中單孤兒藥就高達(dá)28個，占獲批藥物總數(shù)的51%（詳見圖1）。

CDER各年度新藥批準(zhǔn)數(shù)量

圖1 CDER各年度新藥批準(zhǔn)數(shù)量

美國于1983年頒布了《孤兒藥法案》，成為世界上第一個為罕見病藥物立法的國家。之后為了鼓勵罕見病藥物的研發(fā)，美國通過了孤兒藥認(rèn)定（Orphan Drug Designation, ODD）計劃的一系列政策。孤兒藥認(rèn)定（Orphan Drug Designation, ODD）就是FDA 孤兒藥產(chǎn)品開發(fā)辦公室（Office of Orphan Products Development， OOPD) 對符合條件的用于預(yù)防、治療及診斷罕見病的藥物（包括生物制品）授予的一種資格認(rèn)定。

根據(jù)FDA的孤兒藥認(rèn)定數(shù)據(jù)庫資料初步統(tǒng)計，2023年獲得FDA授予的孤兒藥認(rèn)定的創(chuàng)新藥共有376種，其中中國的創(chuàng)新藥占59種（按適應(yīng)癥計）,約占15%（詳見表1）。

國內(nèi)制藥企業(yè)獲得FDA孤兒藥資格認(rèn)定的這些產(chǎn)品，從其適應(yīng)癥領(lǐng)域來看，多集中在腫瘤領(lǐng)域，占比高達(dá)63%（37/59），數(shù)量最多的5個癌種依次是胰腺癌（9項）、胃癌（5項）、惡性膠質(zhì)瘤（4項）、小細(xì)胞肺癌（3項）、膽道癌（2項）。除此之外，肌萎縮側(cè)索硬化癥和亨廷頓舞蹈癥關(guān)注度也較高。

另外，這些產(chǎn)品涵蓋了CAT-T療法、抗體偶聯(lián)藥物（ADC）、雙特異性抗體、基因療法等創(chuàng)新療法。

值得注意的是，有多家公司的多個產(chǎn)品同時被授予孤兒藥資格認(rèn)定。例如博安生物自主開發(fā)的兩款靶向Claudin18.2的在研產(chǎn)品--創(chuàng)新抗體BA1105、創(chuàng)新抗體偶聯(lián)藥物BA1301獲得FDA授予的治療胰腺癌適應(yīng)癥的孤兒藥資格認(rèn)定;Suzhou Immunofoco Biotechnology Co., Ltd（易慕峰）是一家致力于突破實體瘤治療，給患者帶來長期生存獲益的免疫細(xì)胞治療藥物開發(fā)企業(yè)，其自主研發(fā)的靶向EpCAM的自體CAR-T細(xì)胞注射液產(chǎn)品獲得FDA授予的治療胃癌適應(yīng)癥的孤兒藥資格認(rèn)定，時隔不久該產(chǎn)品又被FDA授予孤兒藥資格認(rèn)定，用于治療胰腺癌患者;德琪（杭州）生物自主研發(fā)和開發(fā)的Claudin 18.2抗體偶聯(lián)藥物（ADC）ATG-022獲FDA先后授予的兩項孤兒藥資格認(rèn)定，分別用于治療胃癌及胰腺癌。

表1 2023年獲得FDA授予孤兒藥資格認(rèn)定的中國藥企及產(chǎn)品

NO.	藥品名稱	授予時間	適應(yīng)癥	MAH
1	4-(3-(4-(1H-imidazo[4,5-b]pyridin-5-yl)piperazine-1-carbonyl)-4-fluorobenzyl)phthalazin-1(2H)-one hydrochloride dihydrate	01/09/2023	小細(xì)胞肺癌(SCLC)	Shanghai Yidian Pharmaceutical Technology Development Co., Ltd. 上海壹典醫(yī)藥科技開發(fā)
2	recombinant adeno-associated virus serotype 8 vector encoding human phenylalanine hydroxylase (PAH)	01/09/2023	苯丙酮尿癥（PKU）	NGGT (Suzhou) Biotechnology Co., Ltd.
3	N-(6-(2,6-dichloro-3,5-dimethoxyphenyl)-1H-pyrazolo[3,4-b]pyridin-3-yl)-4-(3,3-dimethylpiperazin-1-yl)benzamide hydrochloride	01/12/2023	胃癌和胃食管交界癌	3D Medicines (Beijing) Co., Ltd
4	(S)-N-(5-(1-(3-ethoxy-4-methoxyphenyl)-2-(methylsulfonyl)ethyl)-4,6-dioxo-5,6-dihydro-4H-thieno[3,4-c]pyrrol-1-yl)acetamide	01/18/2023	白塞病	Ganzhou Hemay Pharmaceutical Co., Ltd.合美醫(yī)藥
5	5¿-(4-fluorophenyl)-3¿-isopropyl-N-(4-(4-methylpiperazin-1-yl) phenyl)-1H,3¿H-[2,4¿-biimidazole]-4-carboxamide acetate	02/01/2023	特發(fā)性肺纖維化	InSilico Medicine Hong Kong Limited英矽智能
6	(-)-B-Elemene（ KH617）	02/14/2023	多型膠質(zhì)母細(xì)胞瘤	Sichuan Honghe Biotechnology Co., Ltd.四川弘合生物科技
7	a humanized, immunoglobulin G subtype 4 anti-Amphiregulin monoclonal antibody	02/16/2023	特發(fā)性肺纖維化	Pulmongene (Hong Kong) Co., Limited普沐(香港)生物科技有限公司
8	allogeneic T cells genetically modified to express anti-B7-H3 chimeric antigen receptor (CAR)	02/16/2023	惡性膠質(zhì)瘤	T-MAXIMUM Pharmaceutical (Suzhou) Co., Ltd.
9	Mefuparib	02/28/2023	膽道癌	Convalife (Shanghai) Co., Ltd.甫康（上海）健康科技
10	siRNA oligonucleotide designed to target and knockdown the expression of SOD1	03/01/2023	肌萎縮側(cè)索硬化癥（ALS）	Ractigen Therapeutics中美瑞康
11	Autologous CD4+/CD8+ T cells transduced with lentiviral vector directed against MAGE-A4 antigen	03/23/2023	IIB至IV期黑色素瘤	TCRx Therapeutics Ltd.科士華生物
12	Humanized, Low-fucose Anti-Claudin 18.2 IgG1 Antibody	03/23/2023	胰腺癌	Suzhou Transcenta Therapeutics Co., Ltd.創(chuàng)勝集團(tuán)
13	3-(5-cyano-4-(cyclopropylamino)pyridin-2-yl)-1-(6-formyl-5-((4-methyl-2-oxopiperazin-1-yl)methyl)pyridin-2-yl)-1-methylurea	03/27/2023	軟組織肉瘤	Wuxi Abbisko Biomedical Technology Co., Ltd.和譽(yù)生物醫(yī)藥
14	a multiple substituted pyrimidoheterocycle small molecule inhibitor that covalently binds to the Switch II pocket of KRAS p.G12C and locks the oncogenic protein in its GDP-bound inactive form	03/27/2023	攜帶KRAS p.G12C突變蛋白的晚期癌癥	D3 Bio (Wuxi) Co., Ltd.德昇濟(jì)
15	A plasmid encoding a rabies virus glycoprotein tag & lysosome-associated membrane glycoprotein 2 fusion protein gene and a mutant huntingtin small interfering RNA	04/10/2023	亨廷頓舞蹈癥	ExoRNA Bioscience Nanjing Co. Ltd.
16	(2-(6-(2-(4-cyclopropylpyrimidin-5-yl)-4-fluorophenoxy)-1,2,4-triazin-5-yl)-2,7-diazaspiro[3.5]nonan-7-yl)((1S,3aR,6aS)-octahydrocyclopenta[c]pyrrol-1-yl)methanone	04/17/2023	急性髓細(xì)胞白血病	BioNova Pharmaceuticals (Shanghai) Limited燁輝醫(yī)藥
17	Edralbrutinib	04/24/2023	視神經(jīng)脊髓炎譜系障礙	Reistone Biopharma Company Limited瑞石生物醫(yī)藥
18	allogeneic umbilical cord mesenchymal stem cells	05/03/2023	眼部移植物抗宿主病	Guangdong ProCapZoom Biosciences Co., Ltd.廣東普羅凱融生物醫(yī)藥
19	Autologous Chimeric Antigen Receptor (CAR) Modified Recombinant T Cells Targeting EGFRvIII	05/09/2023	惡性膠質(zhì)瘤	Beijing DCTY® Biotech Co., Ltd.北京鼎成肽源生物技術(shù)
20	N-[3-fluoro-4-[[1-methyl-6-(1H-pyrazol-4-yl)-1H-indazol-5-yl]oxy]phenyl]-1-(4-fluorophenyl)-1,2-dihydro-6-(1-methylethoxy)-2-oxo-3-pyridinecarboxamide(多靶點激酶抑制劑TSN084)	05/16/2023	用于治療急性髓系白血病	Tyligand Bioscience (Shanghai) Limited泰勵生物
21	Antibody drug conjugate targeting Claudin 18.2 (CLDN 18.2) consisting of a humanized monoclonal antibody against CLDN 18.2 and a conjugated linker-payload VC-MMAE (maleimidocaproyl-valine-citrulline-p-aminobenzylalcohol-Monomethyl Auristatin E)	05/16/2023	胰腺癌	Antengene (Hangzhou) Biologics Co., Ltd.德琪(杭州)生物
22	autologous CD3+ T cell product transduced with transgene constructs expressing anti-human mesothelin (MSLN) CAR and self-secreting anti-human PD-1 nanobody	05/16/2023	間皮瘤	Shanghai Cell Therapy Group Pharmaceutical Technology Co., Ltd上海細(xì)胞治療集團(tuán)
23	antibody drug conjugate targeting Claudin 18.2 (CLDN 18.2) consisting of a humanized monoclonal antibody against CLDN 18.2 and a conjugated linker-payload VC-MMAE (maleimidocaproyl-valine-citrulline-p-aminobenzylalcohol-Monomethyl Auristatin E)	05/22/2023	胃癌	Antengene (Hangzhou) Biologics Co., Ltd德琪(杭州)生物
24	Recombinant humanized anti-MSLN monoclonal antibody- monomethyl auristatin E conjugate	05/22/2023	胰腺癌	RemeGen Co., Ltd榮昌生物
25	chimeric antigen receptor (CAR) modified recombinant T cells targeting B7-H3	06/21/2023	惡性膠質(zhì)瘤	Fuzhou Tcelltech Biological Science and Technology Inc.
26	Pegylated Iron Oxide Nanoparticles	06/26/2023	肝細(xì)胞癌的治療的診斷	MegaPro Biomedical Company, Ltd.巨生生醫(yī)
27	human umbilical cord-derived mesenchymal stem cells	08/09/2023	特發(fā)性肺纖維化	Wuhan Optics Valley Vcanbiopharma Co., Ltd.
28	Autologous synthetic NKG2D receptor armored CLDN18.2 specific CAR-T cell injection	08/15/2023	胃癌	Suzhou Immunofoco Biotechnology Co., Ltd易慕峰
29	Autologous EpCAM specific CAR-T cell injection	08/16/2023	胃癌	Suzhou Immunofoco Biotechnology Co., Ltd易慕峰
30	a genetically modified Salmonella comprising of methioninase	08/28/2023	小細(xì)胞肺癌	Guangzhou Sinogen Pharmaceutical Co., Ltd.廣州華津醫(yī)藥
31	Autologous synthetic NKG2D receptor armored CLDN18.2 specific CAR-T cell injection	08/28/2023	胰腺癌	Suzhou Immunofoco Biotechnology Co., Ltd易慕峰
32	autologous chimeric antigen receptor (CAR) T cells targeting CD7	08/31/2023	急性淋巴細(xì)胞白血病和淋巴細(xì)胞淋巴瘤	Hebei Senlang Biotechnology Co. Ltd.河北森朗生物
33	N( 4{[ 5fluoro7( 2methoxyethoxy) 4quinazolinyl] amino}phenyl)2( 4isopropyl1H1,2,3triazol1yl) acetamide 4-methylbenzenesulfonate (1:1)	09/13/2023	胃腸道間質(zhì)瘤（GIST）	Ningbo NewBay Technology Development Co., Ltd.寧波新灣醫(yī)藥
34	(R)-3-(5,5-dimethyl-4,5-dihydro-1,2,4-oxadiazol-3-yl)-N-(1-(2,3,5-trifluorophenyl) ethyl) pyrazolo[1,5-a] pyrimidin-5-amine hydrate	09/19/2023	ALK陽性、ROS1陽性、NTRK融合陽性或LTK陽性非小細(xì)胞肺癌	TYK Medicines, Inc 同源康
35	Folate-TRPV6-peptide exatecan drug conjugate （CBP-1019）	09/19/2023	胰腺癌	Coherent Biophanna (Hefei) Co., Ltd.同宜醫(yī)藥
36	Human Retinal Pigment Epithelial Cell Injection	09/19/2023	色素性視網(wǎng)膜炎	Eyecure Therapeutics Inc.江蘇艾爾康生物醫(yī)藥科技
37	A Lipid Nanoparticle Suspension of Akt-1 Antisense Oligonucleotide	09/20/2023	肝細(xì)胞癌	Zhejiang Haichang Biotech Co., Ltd.浙江海昶生物醫(yī)藥技術(shù)有限公司
38	((2S,5S)-5-(4-amino-5-(4-(2,3-difluorophenoxy)phenyl)imidazo[5,1-f][1,2,4]triazin-7-yl)tetrahydro-2H-pyran-2-yl)methanol	10/02/2023	用于治療中樞神經(jīng)系統(tǒng)淋巴瘤	Dizal (Jiangsu) Pharmaceutical Co., Ltd.迪哲(江蘇)醫(yī)藥
39	recombinant adeno-associated virus vector serotype 8 carrying the coding sequence of human GBA1 gene	10/05/2023	戈謝病	Lingyi Biotech Co. Ltd凌意（杭州）生物科技
40	Virus-like particle containing Cas9/gRNA ribonucleoprotein targeting the human HTT gene	10/05/2023	亨廷頓舞蹈癥	Shanghai BDgene Co., Ltd.上海本島基因
41	adeno-associated virus (AAV) vector-based gene therapy delivering a therapeutic transgene of RJK002	10/16/2023	肌萎縮側(cè)索硬化癥	Rejukon Biopharm Inc.瑞吉康生物醫(yī)藥有限公司
42	recombinant humanized anti-interleukin 36R monoclonal antibody	10/16/2023	泛發(fā)性膿皰型銀屑病	Shanghai Huaota Biopharmaceutical Co., Ltd.上海華奧泰生物藥業(yè)
43	N-(2-(4-Fluorophenyl)-5,7-dimethyl-1,2,3,4-tetrahydro isoquinolin-6-yl)-2-(1-methylcyclopropyl) acetamide	10/18/2023	肌萎縮側(cè)索硬化癥	Shanghai Zhimeng Biopharma, Inc上海摯盟醫(yī)藥科技
44	Recombinant human signal regulating protein alpha (SIRPalpha) fragment crystallizable (Fc)-fusion protein	11/07/2023	慢性粒單核細(xì)胞白血?。–MML）	ImmuneOnco Biopharmaceuticals (Shanghai) Inc.宜明昂科
45	Recombinant human IgG1 anti-B7H3 monoclonal antibody conjugated to topoisomerase I inhibitor YL0010014	11/16/2023	食管癌	MediLink Therapeutics蘇州宜聯(lián)生物醫(yī)藥
46	Allogenic spinal motor neuron precursor cell	11/22/2023	肌萎縮側(cè)索硬化癥（ALS）	XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd士澤生物醫(yī)藥(蘇州)
47	Epirubicin	11/28/2023	惡性膠質(zhì)瘤	Beijing Inno Medicine Co., Ltd.茵諾醫(yī)藥
48	Chaenomelis Fructus Extract	11/29/2023	BAG3相關(guān)肌原纖維肌病	Centre for Chinese Herbal Medicine Drug Development Limited中藥創(chuàng)新研發(fā)中心
49	a bispecific antibody composed of a Light Chain and a Heavy Chain pair targeting HER2, and a Light Chain and a Heavy Chain pair targeting CD47	12/07/2023	胃癌癥患者的治療，包括胃食管交界處癌癥	D3 Bio (Wuxi) Co., Ltd.德昇濟(jì)
50	Tetrahydro-2H-pyran-4-yl 7-(2-(cyclopropanecarboxamido)- imidazo[1,2-a]pyridin-6-yl)-2,3-dihydro-1H-pyrido[2,3-b][1,4]oxazine-1-carboxylate hydrochloride	12/11/2023	急性移植物抗宿主病	Accro Bioscience (Suzhou) Limited愛科諾生物醫(yī)藥(蘇州)有限公司
51	(-)-Huperzine A石杉堿甲	12/12/2023	重癥肌無力	Wanbangde Pharmaceutical Group Co, Ltd.萬邦德制藥
52	recombinant humanized anti-human tissue factor monoclonal antibody conjugated with a cytotoxic small molecule MMAE	12/12/2023	胰腺癌	Shanghai Miracogen Inc.上海美雅珂生物
53	trastuzumab-rezetecan	12/14/2023	膽道癌	Jiangsu Hengrui Pharmaceuticals Co., Ltd. 恒瑞醫(yī)藥
54	recombinant fusion protein composed of a recombinant human arylsulfatase A (rhARSA) fused with a variable domain of the heavy chain of heavy chain-only antibody fragment targeting human transferrin	12/15/2023	異染性腦白質(zhì)營養(yǎng)不良	Linno Pharmaceuticals, Inc.領(lǐng)諾醫(yī)藥
55	an IgG1(Immunoglobulin G1) subclass antibody targeting CCR8(C-C Motif Chemokine Receptor 8)	12/18/2023	胰腺癌	LaNova Medicines Limited禮新醫(yī)藥
56	Adeno-associated viruses 8 (AAV8)-based gene therapy vector that expresses B domain-deleted human factor VIII under the control of human liver-specific promoters	12/19/2023	血友病A	Gritgen Therapeutics Co., Ltd.華毅樂健
57	anti-Claudin18.2 monoclonal antibody conjugated with Duostatin-5 payload	12/21/2023	胰腺癌	Shandong Boan Biotechnology Co., Ltd.博安生物
58	Human monoclonal IgG1 antibody against the isoform 2 of human Claudin 18 protein (Claudin 18.2)	12/21/2023	胰腺癌	Shandong Boan Biotechnology Co., Ltd.博安生物
59	Dual recombinant adeno-associated viral vectors encoding the human otoferlin gene	12/21/2023	耳啡肽介導(dǎo)的聽力損失	Shanghai Refreshgene Therapeutics Co., Ltd.上海鼎新基因科技

分析

從上面這些數(shù)據(jù)看，國內(nèi)創(chuàng)新藥企業(yè)對美國孤兒藥的政策很熟悉，并且充分運(yùn)用。對于研發(fā)成本高啟的美國醫(yī)藥市場，可以通過孤兒藥資格認(rèn)定這條途徑，開啟合適的臨床試驗并控制研發(fā)費(fèi)用，對于創(chuàng)新藥公司是一條不錯的開辟途徑。

參考資料：

1-FDA官網(wǎng)

2-FDA《2023年新藥治療審批報告》

作者簡介：zhulikou431，高級工程師、PDA會員、ISPE會員、ECA會員、PQRI會員、資深無菌GMP專家，在無菌工藝開發(fā)和驗證、藥品研發(fā)和注冊、CTD文件撰寫和審核、法規(guī)審計、國際認(rèn)證、國際注冊、質(zhì)量體系建設(shè)與維護(hù)領(lǐng)域，以及無菌檢驗、環(huán)境監(jiān)控等領(lǐng)域皆具有較深造詣。近幾年開始著力關(guān)注制藥宏觀領(lǐng)域趨勢分析和制藥企業(yè)并購項目的風(fēng)險管理工作。

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zhulikou431

高級工程師、PDA會員、ISPE會員、ECA會員、PQRI會員、資深無菌GMP專家，在無菌工藝開發(fā)和驗證、藥品研發(fā)和注冊、CTD文件撰寫和審核、法規(guī)審計、國際認(rèn)證、國際注冊、質(zhì)量體系建設(shè)與維護(hù)領(lǐng)域，以及無菌檢驗、環(huán)境監(jiān)控等領(lǐng)域皆具有較深造詣。近幾年開始著力關(guān)注制藥宏觀領(lǐng)域趨勢分析和制藥企業(yè)并購項目的風(fēng)險管理工作。

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