Widening the path and window of opportunity for FDA approval of non-vitamin K oral anticoagulant specific antidotes and reversal agents

设为首页

收藏本站

网站地图 | English | 公务邮箱

About the library

Background
History
Leadership
Organization

Readers' Guide

Opening Hours
Collections
Help Via Email

Publications

Electronic Information Resources

Widening the path and window of opportunity for FDA approval of non-vitamin K oral anticoagulant specific antidotes and reversal agents

详细信息查看全文

作者：Sunny Patel ; Dylan Steen
关键词：Novel oral anticoagulant ; Reversal agent ; Antidote ; Drug approval
刊名：Journal of Thrombosis and Thrombolysis
出版年：2016
出版时间：February 2016
年：2016
卷：41
期：2
页码：285-292
全文大小：577 KB
参考文献：1.Sarich TC, Seltzer JH, Berkowitz SD et al (2015) Novel oral anticoagulants and reversal agents: considerations for clinical development. Am Heart J 169(6):751–757CrossRef PubMed
2.Siegal DM (2015) Managing target-specific oral anticoagulant associated bleeding including an update on pharmacological reversal agents. J Thromb Thrombolysis 39(3):395–402. doi:10.1007/s11239-015-1167-9 CrossRef PubMed
3.Battinelli EM (2011) Reversal of new oral anticoagulants. Circulation 124(14):1508–1510. doi:10.1161/CIRCULATIONAHA.111.054510 CrossRef PubMed
4.Gomez-Outes A, Suarez-Gea ML, Lecumberri R et al (2014) Specific antidotes in development for reversal of novel anticoagulants: a review. Recent Pat Cardiovasc Drug Discov 9(1):2–10CrossRef PubMed
5.ClinicalTrials.gov (2015) Reversal of dabigatran anticoagulant effect with idarucizumab. U.S. National Library of Medicine. https://clinicaltrials.gov/ct2/show/NCT02104947?term=Idarucizumab&rank=1 . Accessed 18 July 2015
6.ClinicalTrials.gov (2015) A study in older subject to evaluate the safety and ability of andexanet alfa to reverse the anticoagulation effect of rivaroxaban. U.S. National Library of Medicine. https://clinicaltrials.gov/ct2/show/NCT02220725?term=Andexanet+alfa&rank=1 . Accessed 18 July 2015
7.ClinicalTrials.gov (2015) A study in older subjects to evaluate the safety and ability of andexanet alfa to reverse the anticoagulation effect of apixaban. U.S. National Library of Medicine. https://clinicaltrials.gov/ct2/show/NCT02207725?term=Andexanet+alfa&rank=2 . Accessed 18 July 2015
8.ClinicalTrials.gov (2015) Recombinant factor Xa inhibitor antidote. U.S. National Library of Medicine. https://clinicaltrials.gov/ct2/show/NCT02329327?term=Andexanet+alfa&rank=3 . Accessed 18 July 2015
9.ClinicalTrials.gov (2015) Study of PER977 administered to subjects with steady state edoxaban dosing and re-anticoagulation with edoxaban. U.S. National Library of Medicine. https://clinicaltrials.gov/ct2/show/NCT02207257?term=PER977&rank=3 . Accessed 18 July 2015
10.ClinicalTrials.gov (2015) Evaluation of the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of PER977 following heparin. U.S. National Library of Medicine. https://clinicaltrials.gov/ct2/show/NCT02206087?term=PER977&rank=2 . Accessed 18 July 2015
11.Roden DM, Temple R (2005) The US food and drug administration cardiorenal advisory panel and the drug approval process. Circulation 111(13):1697–1702CrossRef PubMed
12.Katz R (2004) FDA: evidentiary standards for drug development and approval. NeuroRx 1(3):307–316. doi:10.1602/neurorx.1.3.307 PubMedCentral CrossRef PubMed
13.Food and Drug Administration (1998) Guidance for industry providing clinical evidence of effectiveness for human drug and biological products. US Department of Health and Human Services. http://www.fda.gov/downloads/Drugs/…/Guidances/ucm078749.pdf . Accessed 06 June 2015
14.Califf RM (2013) Biomarkers, putative surrogates, surrogates, and decision making. Circ Cardiovasc Imaging 6(1):6–7. doi:10.1161/CIRCIMAGING.112.982538 CrossRef PubMed
15.Karsdal MA, Henriksen K, Leeming DJ et al (2009) Biochemical markers and the FDA critical path: how biomarkers may contribute to the understanding of pathophysiology and provide unique and necessary tools for drug development. Biomarkers 14(3):181–202. doi:10.1080/13547500902777608 CrossRef PubMed
16.Baglin T (2013) The role of the laboratory in treatment with new oral anticoagulants. J Thromb Haemost 11(1):122–128. doi:10.1111/jth.12227 CrossRef PubMed
17.Lesko LJ, Atkinson AJ Jr (2001) Use of biomarkers and surrogate endpoints in drug development and regulatory decision making: criteria, validation, strategies. Annu Rev Pharmacol Toxicol 41:347–366CrossRef PubMed
18.Rajman I (2008) PK/PD modelling and simulations: utility in drug development. Drug Discov Today 13(7–8):341–346. doi:10.1016/j.drudis.2008.01.003 CrossRef PubMed
19.Reilly PA, Lehr T, Haertter S et al (2014) The effect of dabigatran plasma concentrations and patient characteristics on the frequency of ischemic stroke and major bleeding in atrial fibrillation patients: the RE-LY Trial (Randomized Evaluation of Long-Term Anticoagulation Therapy). J Am Coll Cardiol 63(4):321–328. doi:10.1016/j.jacc.2013.07.104 CrossRef PubMed
20.Murphy L (2015) FDA grants priority review to Boehringer Ingelheim’s biologics license application for idarucizumab*. Boehringer Ingelheim Pharmaceuticals. http://us.boehringer-ingelheim.com/news_events/press_releases/press_release_archive/2015/4-23-2015-fda-grants-priority-review-boehringer-ingelheims-biologics-license-application-idarucizumab.html . Accessed 10 June 2015
21.Portola Pharmaceuticals (2015) Portola pharmaceuticals initiates phase 4 study to support accelerated approval of andexanet alfa: its breakthrough-designated factor Xa inhibitor antidote. Portola Pharmaceuticals, Inc. http://investors.portola.com/phoenix.zhtml?c=198136&p=irol-newsArticle&ID=2006796 . Accessed 28 July 2015
22.Steiner, S (2015) Perosphere receives FDA fast track designation for investigational anticoagulant reversal agent PER977. Perosphere Inc. http://perosphere.com/documents/PerosphereFDAFastTrack.pdf . Accessed 27 July 2015
23.Steiner S (2015) Perosphere and Daiichi Sankyo enter into a clinical trial collaboration agreement for phase 3 studies of PER977 to investigate reversal of the anticoagulant activity of the investigational factor Xa-inhibitor Edoxaban. Perosphere Inc. http://perosphere.com/content/media/documents/PerosphereandDaiichiSankyoEnterintoaClinicalTrialAgreementforPhase3.pdf . Accessed 27 July 2015
24.Food and Drug Administration (2014) Guidance for industry: expedited programs for serious conditions—drugs and biologics. US Department of Health and Human Services. http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm358301.pdf . Accessed 14 June 2015
25.Woodcock J, Woosley R (2008) The FDA critical path initiative and its influence on new drug development. Annu Rev Med 59:1–12. doi:10.1146/annurev.med.59.090506.155819 CrossRef PubMed
26.Muller PY, Brennan FR (2009) Safety assessment and dose selection for first-in-human clinical trials with immunomodulatory monoclonal antibodies. Clin Pharmacol Ther 85(3):247–258. doi:10.1038/clpt.2008.273 CrossRef PubMed
27.Tenaerts P, Madre L, Archdeacon P et al (2014) The clinical trials transformation Initiative: innovation through collaboration. Nat Rev Drug Discov 13(11):797–798. doi:10.1038/nrd4442 CrossRef PubMed
28.Food and Drug Administration (2010) Guidance for industry: adaptive design clinical trials for drugs and biologics. US Department of Health and Human Services. http://www.fda.gov/downloads/Drugs/…/Guidances/ucm201790.pdf . Accessed 11 July 2015
29.Lauer MS, D’Agostino RBS (2013) The randomized registry trial: the next disruptive technology in clinical research? N Engl J Med 369(17):1579–1581. doi:10.1056/NEJMp1310102 CrossRef PubMed
30.James S, Rao SV, Granger CB (2015) Registry-based randomized clinical trials: a new clinical trial paradigm. Nat Rev Cardiol 12(5):312–316. doi:10.1038/nrcardio.2015.33 CrossRef PubMed
31.Frobert O, Lagerqvist B, Gudnason T et al (2010) Thrombus Aspiration in ST-Elevation myocardial infarction in Scandinavia (TASTE trial). A multicenter, prospective, randomized, controlled clinical registry trial based on the Swedish angiography and angioplasty registry (SCAAR) platform. Study design and rationale. Am Heart J 160(6):1042–1048. doi:10.1016/j.ahj.2010.08.040 CrossRef PubMed
32.Lagerqvist B, Frobert O, Olivecrona GK et al (2014) Outcomes 1 year after thrombus aspiration for myocardial infarction. N Engl J Med 371(12):1111–1120. doi:10.1056/NEJMoa1405707 CrossRef PubMed
作者单位：Sunny Patel (1)
Dylan Steen (1) (2)

1. University of Cincinnati College of Medicine, Cincinnati, OH, 45267, USA
2. Division of CV Health and Disease, UC Heart, Lung, and Vascular Institute, Cardiovascular Center, University of Cincinnati College of Medicine, 231 Albert Sabin Way, Rm 3923, Cincinnati, OH, 45267, USA
刊物类别：Medicine
刊物主题：Medicine & Public Health
Cardiology
Hematology
出版者：Springer Netherlands
ISSN：1573-742X

文摘

There remains a need for safe, immediately effective, and easy to administer antidotes for patients taking novel oral anticoagulants (NOACs) in the settings of major bleeding, need for emergency surgery, and accidental overdose. We review considerations for the successful safety and effectiveness evaluation of potential antidotes currently under development. These compounds are in expedited regulatory approval programs aimed at accelerating the preclinical and clinical evaluation and approval processes for treatments of serious conditions. We review the features of these expedited programs as well as the FDA’s efforts to broadly advance the efficiency of drug development and increase the number of new compounds brought to market. The critical path initiative and regulatory science initiative have resulted in numerous successful programs to address current challenges such as a paucity of validated biomarkers and surrogate endpoints as well as unreliable animal models of toxicity. The FDA has also advocated for increased use of pharmacokinetic/pharmacodynamic modeling and adaptive trial design. These efforts foster collaboration between academia, industry and the public sector across interdisciplinary sciences and may continue to widen the pathway for NOAC-specific reversal agents and other novel compounds. Keywords Novel oral anticoagulant Reversal agent Antidote Drug approval