2023년 미국 FDA 55종의 신약 허가승인

미국에서 의약품을 판매하기 위해서는 필수적으로 미국 FDA의 심사를 거쳐서 승인을 받아야 한다.

2023년 한해동안 미국 FDA의 의약품 평가연구센터(CDER: Center for Drug Evaluation and Research)는 총 55종의 신약을 승인하였다. 2022년 허가신약 37건 대비 약 50% 증가한 수치이다.

2023년 승인된 55건의 신약중 28건(51%)이 희귀의약품 지정(Orphan Drug Designation, ODD)을 통하여 승인을 받았으며, 20건(36%)이 혁신신약 (first-in-class)으로 분류되었다.

 

<연도별 FDA 신약허가 건수: 1994~2023>

NMEs: New Molecular Entities(신규 저분자 합성신약), BLAs: Biologics License Applications(생물의약품 품목허가)



 

2023년 승인된 55건의 신약에 대하여

1. 적응증별로 보면,

항암제(Oncology ): 13건, 뇌 또는 신경관련 의약품(Neurology): 9건, 감염병 및 혈액관련 의약품(Infectious diseases & Haematology): 각 5건의 순서로 나타났다.

 

2. 약물유형(Modality)별로 보면,

저분자 합성신약: 34건,

단일클론항체신약 (monoclonal antibody): 12건,

단백질 의약품 (효소대체제, 호르몬, 융합단백질 등): 5건

올리고뉴클레오타이드 (siRNA, antisense ologonucleotides, RNA aptamer 등): 4건

 

3. 신속심사 프로그램 활용

또한 대부분 신약허가신청은 신속하게 심사를 받기 위하여 ‘신속심사프로그램’ 제도를 활용하여 진행되었다. (55건중 49건, 89%).

‘신속심사프로그램’을 이용한 허가심사유형별로 보면

1. 1. 패스트 트랙(Fast track): 25/55 (45%)
   2. 획기적 치료제 지정(Breakthrough Therapy Designation): 9/55 (16%)
   3. 우선 심사(Priority review): 31/55 (56%)
   4. 신속심사(Accelerated approval): 9/55 (16%)

 

<2023년 FDA 승인 55종 신약 목록>

순번	성분명 (제품명)	개발사	작용기전(Properties)	적응증(Indication)
1	Lecanemab (Leqembi)	Eisai/Biogen	Amyloid-β-targeted mAb	Alzheimer disease
2	Bexagliflozin (Brenzavvy)	Theracosbio	SGLT2 inhibitor	Glycaemic control in type 2 diabetes mellitus
3	Pirtobrutinib (Jaypirca)	Loxo/Eli Lilly	BTK inhibitor	Mantle cell lymphoma
4	Elacestrant (Orserdu)	Stemline	ER antagonist	ER-positive, HER2-negative, ESR1-mutant breast cancer
5	Daprodustat (Jesduvroq)	GSK	HIF-PH inhibitor	Anaemia caused by CKD for adults on dialysis
6	Velmanase alfa (Lamzede)	Chiesi	Recombinant α-mannosidase	Non-CNS manifestations of α-mannosidosis
7	Sparsentan (Filspari)	Travere	Endothelin and angiotensin II receptor antagonist	Proteinuria in primary IgA nephropathy
8	Omaveloxolone (Skyclarys)	Reata/Biogen	Mechanism unknown, NRF2 activator	Friedrich’s ataxia
9	Zavegepant (Zavzpret)	Pfizer	CGRP receptor antagonist	Migraine
10	Trofinetide (Daybue)	Acadia	Mechanism unknown	Rett syndrome
11	Retifanlimab (Zynyz)	Incyte	PD1-targeted mAb	Merkel cell carcinoma
12	Rezafungin (Rezzayo)	Cidara	Echinocandin antifungal	Candidemia and invasive candidiasis
13	Leniolisib (Joenja)	Pharming	PI3Kδ inhibitor	Activated PI3Kδ syndrome
14	Tofersen (Qalsody)	Biogen	SOD1-targeted ASO	SOD1 amyotrophic lateral sclerosis
15	Pegunigalsidase alfa (Elfabrio)	Chiesi	PEGylated recombinant α-galactosidase Α	Fabry disease
16	Fezolinetant (Veozah)	Astellas	Neurokinin 3 receptor antagonist	Hot flashes caused by menopause
17	Perfluorohexyloctane (Miebo)	Bausch + Lomb	Semifluorinated alkane	Dry eye disease
18	Epcoritamab (Epkinly)	Genmab/AbbVie	CD20 × CD3 T-cell engager	DLBCL and high-grade B-cell lymphoma
19	Sulbactam/durlobactam (Xacduro)	Entasis	β-lactam antibacterial plus a β-lactamase inhibitor	Hospital-acquired and ventilator-associated bacterial pneumonia caused by susceptible ABC
20	Nirmatrelvir/ritonavir (Paxlovid)	Pfizer	SARS-CoV-2 main protease inhibitor plus a CYP3A inhibitor	Mild-to-moderate COVID-19
21	Flotufolastat F18 (Posluma)	Blue Earth	Radioactive diagnostic agent	PET imaging in prostate cancer
22	Sotagliflozin (Inpefa)	Lexicon	SGLT1/2 inhibitor	Heart failure
23	Glofitamab (Columvi)	Genentech	CD20 × CD3 T-cell engager	DLBLC or large B-cell lymphoma
24	Ritlecitinib (Litfulo)	Pfizer	JAK3 inhibitor	Alopecia areata
25	Rozanolixizumab (Rystiggo)	UCB	FcRn-targeted mAb	AChR- or MuSK-antibody-positive gMG
26	Somatrogon (Ngenla)	Pfizer	Human growth hormone analogue	Growth hormone deficiency
27	Nirsevimab (Beyfortus)	AstraZeneca	RSV F protein-targeted mAb	RSV lower respiratory tract disease
28	Quizartinib (Vanflyta)	Daiichi Sankyo	FLT3 kinase inhibitor	AML
29	Lotilaner (Xdemvy)	Tarsus	Ectoparasiticide	Demodex blepharitis
30	Zuranolone (Zurzuvae)	Sage	GABAAreceptor PAM	Postpartum depression
31	Avacincaptad pegol (Izervay)	Iveric/Astellas	C5-targeted aptamer	Geographic atrophy secondary to AMD
32	Talquetamab (Talvey)	Janssen	GPRC5D × CD3 T-cell engager	Multiple myeloma
33	Elranatamab (Elrexfio)	Pfizer	BCMA × CD3 T-cell engager	Multiple myeloma
34	Palovarotene (Sohonos)	Ipsen	Retinoic acid receptor agonist	Fibrodysplasia ossificans progressiva
35	Pozelimab (Veopoz)	Regeneron	C5-targeted mAb	CHAPLE disease
36	Motixafortide (Aphexda)	Biolinerx	CXCR4 inhibitor	Hematopoietic stem cell mobilization for autologous transplantation in multiple myeloma
37	Momelotinib (Ojjaara)	GSK	JAK1/2, ALK2 inhibitor	Myelofibrosis in adults with anaemia
38	Gepirone (Exxua)	Fabre-Kramer	5HT1Areceptor agonist	Major depressive disorder
39	Cipaglucosidase alfa (Pombiliti)	Amicus	Recombinant α-glucosidase	Pompe disease
40	Nedosiran (Rivfloza)	Novo Nordisk	LDHA-targeted siRNA	Primary hyperoxaluria type 1
41	Etrasimod (Velsipity)	Pfizer	S1P receptor modulator	Ulcerative colitis
42	Zilucoplan (Zilbrysq)	UCB	Complement C5 inhibitor	AChR-antibody positive gMG
43	Bimekizumab (Bimzelx)	UCB	IL-17A/F-targeted mAb	Plaque psoriasis
44	Vamorolone (Agamree)	Santhera	Corticosteroid	Duchenne muscular dystrophy
45	Mirikizumab (Omvoh)	Eli Lilly	IL-23-targeted mAb	Ulcerative colitis
46	Toripalimab (Loqtorzi)	Coherus	PD1-targeted mAb	Nasopharyngeal carcinoma
47	Fruquintinib (Fruzaqla)	Takeda	VEGFR1/2/3 kinase inhibitor	Colorectal cancer
48	Taurolidine/heparin (Defencath)	Cormedix	Thiadiazinane antimicrobial plus an anticoagulant	Incidence of catheter-related bloodstream infections
49	Repotrectinib (Augtyro)	Bristol Myers Squibb	ROS1 and TRK kinase inhibitor	ROS1-positive NSCLC
50	Efbemalenograstim alfa (Ryzneuta)	Evive	Recombinant leukocyte growth factor	Neutropenia
51	Capivasertib (Truqap)	AstraZeneca	AKT kinase inhibitor	Breast cancer
52	Nirogacestat (Ogsiveo)	Springworks	γ-secretase inhibitor	Desmoid tumours
53	Iptacopan (Fabhalta)	Novartis	Complement factor B inhibitor	Paroxysmal nocturnal haemoglobinuria
54	Birch triterpenes (Filsuvez)	Chiesi	Mechanism unknown	Epidermolysis bullosa
55	Eplontersen (Wainua)	Ionis/AstraZeneca	TTR-targeted ASO	hATTR with polyneuropathy

 

55종 신약별 상세한 적응증, 작용기전, 약물유형, 약물구조에 대한 정보는 아래 참조

2023년 1월~4월 사이에 승인된 14종의 신약(순번 1~14)에 대한 정보(바로가기)

2023년 5월~6월 사이에 승인된 12종의 신약(순번 15~26)에 대한 정보(바로가기)

2023년 7월~8월 사이에 승인된 9종의 신약(순번 27~35)에 대한 정보(바로가기)

2023년 9월~12월 사이에 승인된 20종의 신약(순번 36~55)에 대한 정보(바로가기)

작성: 이현규 (한국화학연구원)

<참고자료>

1. FDA’s Center for Drug Evaluation and Research’s (CDER) 13th annual report, Advancing Health Through Innovation: New Drug Therapy Approvals.(바로가기)
2. 2023 FDA approvals, Nature Reviews Drug Discovery, 02 January 2024.(바로가기)