Benefit-risk assessment for brincidofovir for the treatment of smallpox: US Food and Drug Administration's Evaluation

被引：56

作者：

Chan-Tack, Kirk ^{[1
]}

Harrington, Patrick ^{[1
]}

Bensman, Timothy ^{[2
]}

Choi, Su-Young ^{[2
]}

Donaldson, Eric ^{[1
]}

O'Rear, Julian ^{[1
]}

McMillan, David ^{[3
]}

Myers, Laine ^{[3
]}

Seaton, Mark ^{[3
]}

Ghantous, Hanan ^{[3
]}

Cao, Yu ^{[4
]}

Valappil, Thamban ^{[4
]}

Birnkrant, Debra ^{[1
]}

Struble, Kimberly ^{[1
]}

机构：

[1] US FDA, Div Antivirals, Off Infect Dis, Ctr Drug Evaluat & Res, Silver Spring, MD 20993 USA

[2] US FDA, Off Translat Sci, Off Clin Pharmacol, Div Clin Pharmacol 4,Ctr Drug Evaluat & Res, Silver Spring, MD 20993 USA

[3] US FDA, Div Pharmacol Toxicol Infect Dis, Off Infect Dis, Ctr Drug Evaluat & Res, Silver Spring, MD 20993 USA

[4] US FDA, Div Biometr 4, Off Biostat, Ctr Drug Evaluat & Res, Silver Spring, MD 20993 USA

来源：

ANTIVIRAL RESEARCH | 2021年 / 195卷

关键词：

Variola virus; Smallpox; Antiviral; Animal rule; THERAPEUTIC INTERVENTION; EFFICACY; MOUSEPOX; DISEASE; CMX001; MODEL; BIOMARKERS;

D O I：

10.1016/j.antiviral.2021.105182

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

The development and approval of brincidofovir for the treatment of smallpox, a disease that was eradicated from the world over 40 years ago, has resulted in the second antiviral approved via the Medical Countermeasure Initiative (MCMi) to combat this disease. Approval of brincidofovir required a unique regulatory approach based on the FDA Animal Rule, and development was supported by many years of research and collaboration among academic investigators, the pharmaceutical industry and multiple government agencies. This article summarizes the FDA regulatory pathway and describes the challenges involved.

引用

页数：5

共 21 条

[1]

[Anonymous], 2015, FDA Animal Rule

[2]

[Anonymous], 2011, ANT DRUGS ADV COMM M

[3] Current concepts - Diagnosis and management of smallpox [J].

Breman, JG ;

Henderson, DA .

NEW ENGLAND JOURNAL OF MEDICINE, 2002, 346 (17) :1300-1308

[4] Assessing a drug for an eradicated human disease: US Food and Drug Administration review of tecovirimat for the treatment of smallpox [J].

Chan-Tack, Kirk M. ;

Harrington, Patrick R. ;

Choi, Su-Young ;

Myers, Laine ;

O'Rear, Julian ;

Seo, Shirley ;

McMillan, David ;

Ghantous, Hanan ;

Birnkrant, Debra ;

Sherwat, Adam, I .

LANCET INFECTIOUS DISEASES, 2019, 19 (06) :E221-E224

[5] Poxvirus interleukin-4 expression overcomes inherent resistance and vaccine-induced immunity: Pathogenesis, prophylaxis, and antiviral therapy [J].

Chen, Nanhai ;

Bellone, Clifford J. ;

Schriewer, Jill ;

Owens, Gelita ;

Fredrickson, Torgny ;

Parker, Scott ;

Buller, R. Mark L. .

VIROLOGY, 2011, 409 (02) :328-337

[6]

Chimerix Inc, 2021, TEMBEXA PACK INS

[7] Brincidofovir for Asymptomatic Adenovirus Viremia in Pediatric and Adult Allogeneic Hematopoietic Cell Transplant Recipients: A Randomized Placebo-Controlled Phase II Trial [J].

Grimley, Michael S. ;

Chemaly, Roy F. ;

Englund, Janet A. ;

Kurtzberg, Joanne ;

Chittick, Gregory ;

Brundage, Thomas M. ;

Bae, Andrew ;

Morrison, Marion E. ;

Prasad, Vinod K. .

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2017, 23 (03) :512-521

[8] Efficacy of delayed brincidofovir treatment against a lethal rabbitpox virus challenge in New Zealand White rabbits [J].

Grossi, Irma M. ;

Foster, Scott A. ;

Gainey, Melicia R. ;

Krile, Robert T. ;

Dunn, John A. ;

Brundage, Thomas ;

Khouri, Jody M. .

ANTIVIRAL RESEARCH, 2017, 143 :278-286

[9] A Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial of Oral Brincidofovir for Cytomegalovirus Prophylaxis in Allogeneic Hematopoietic Cell Transplantation [J].

Marty, Francisco M. ;

Winston, Drew J. ;

Chemaly, Roy F. ;

Mullane, Kathleen M. ;

Shore, Tsiporah B. ;

Papanicolaou, Genovefa A. ;

Chittick, Greg ;

Brundage, Thomas M. ;

Wilson, Chad ;

Morrison, Marion E. ;

Foster, Scott A. ;

Nichols, W. Garrett ;

Boeckh, Michael J. ;

Beguin, Yves ;

Poire, Xavier ;

Bambace, Nadia ;

Rotstein, Coleman ;

Wall, Donna ;

Alangaden, George ;

Avery, Robin ;

Boeckh, Michael ;

Boyer, Michael ;

Brunvand, Mark ;

Chhabra, Saurabh ;

Chandrasekar, Pranatharthi ;

Chemaly, Roy ;

Clark, Nina ;

Derman, Olga ;

Flomenberg, Phyllis ;

Green, Jaime ;

Grimley, Michael ;

Gutman, Jonathan ;

High, Kevin ;

Ho, Richard ;

Hoda, Daanish ;

Holland, Kent ;

Islas-Ohlmayer, Miguel ;

Ison, Michael ;

Kaul, Daniel ;

Khan, Cyrus ;

Langston, Amelia ;

Levine, James ;

Long, Gwynn ;

Marty, Francisco ;

Meier, Jeffrey ;

Morris, Michele ;

Mullane, Kathleen ;

Papanicolaou, Genovefa ;

Rowley, Scott ;

Scigliano, Eileen .

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2019, 25 (02) :369-381

[10] CMX001 to Prevent Cytomegalovirus Disease in Hematopoietic-Cell Transplantation [J].

Marty, Francisco M. ;

Winston, Drew J. ;

Rowley, Scott D. ;

Vance, Estil ;

Papanicolaou, Genovefa A. ;

Mullane, Kathleen M. ;

Brundage, Thomas M. ;

Robertson, Alice T. ;

Godkin, Susan ;

Mommeja-Marin, Herve ;

Boeckh, Michael .

NEW ENGLAND JOURNAL OF MEDICINE, 2013, 369 (13) :1227-1236

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