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Clofazimine As a Treatment for Multidrug-Resistant Tuberculosis: a Review
Scientia Pharmaceutica Review Clofazimine as a Treatment for Multidrug-Resistant Tuberculosis: A Review Rhea Veda Nugraha 1 , Vycke Yunivita 2 , Prayudi Santoso 3, Rob E. Aarnoutse 4 and Rovina Ruslami 2,* 1 Department of Biomedical Sciences, Faculty of Medicine, Universitas Padjadjaran, Bandung 40161, Indonesia; [email protected] 2 Division of Pharmacology and Therapy, Department of Biomedical Sciences, Faculty of Medicine, Universitas Padjadjaran, Bandung 40161, Indonesia; [email protected] 3 Department of Internal Medicine, Faculty of Medicine, Universitas Padjadjaran—Hasan Sadikin Hospital, Bandung 40161, Indonesia; [email protected] 4 Department of Pharmacy, Radboud University Medical Center, Radboud Institute for Health Sciences, 6255HB Nijmegen, The Netherlands; [email protected] * Correspondence: [email protected] Abstract: Multidrug-resistant tuberculosis (MDR-TB) is an infectious disease caused by Mycobac- terium tuberculosis which is resistant to at least isoniazid and rifampicin. This disease is a worldwide threat and complicates the control of tuberculosis (TB). Long treatment duration, a combination of several drugs, and the adverse effects of these drugs are the factors that play a role in the poor outcomes of MDR-TB patients. There have been many studies with repurposed drugs to improve MDR-TB outcomes, including clofazimine. Clofazimine recently moved from group 5 to group B of drugs that are used to treat MDR-TB. This drug belongs to the riminophenazine class, which has lipophilic characteristics and was previously discovered to treat TB and approved for leprosy. This review discusses the role of clofazimine as a treatment component in patients with MDR-TB, and Citation: Nugraha, R.V.; Yunivita, V.; the drug’s properties. -
Inhaled Anti-Tubercular Therapy: Dry Powder Formulations, Device And
UNI SYDNEY LOGO INHALED ANTI-TUBERCULAR THERAPY: DRY POWDER FORMULATIONS, DEVICE AND TOXICITY CHALLENGES Thaigarajan Parumasivam A thesis submitted in fulfillment of the requirements for the degree of Doctor of Philosophy Faculty of Pharmacy The University of Sydney 2017 STATEMENT OF AUTHENTICITY This thesis is submitted to the University of Sydney in fulfilment of the requirements for the Degree of Doctor of Philosophy. The work described was carried out in the Faculty of Pharmacy and Centenary Institute under the supervision of Professor Hak-Kim Chan and Professor Warwick Britton The work presented in this thesis, is to the best of my knowledge and belief, original except as acknowledged in the text. The contributions of all co-authors in publications included in the body of the thesis have been declared, signed by each co-author and attached as an appendix. I hereby declare that I have not previously or concurrently submitted this material, either in full or in part, for a degree at this or any other institution. Thaigarajan Parumasivam Oct 2016 ii CONTENTS Acknowledgement ................................................................................................................. viii Glossary .................................................................................................................................... x Thesis abstract .......................................................................................................................... 1 Chapter 1 ....................................................................................................................... -
Treatment of Drug-Resistant Tuberculosis an Official ATS/CDC/ERS/IDSA Clinical Practice Guideline Payam Nahid, Sundari R
AMERICAN THORACIC SOCIETY DOCUMENTS Treatment of Drug-Resistant Tuberculosis An Official ATS/CDC/ERS/IDSA Clinical Practice Guideline Payam Nahid, Sundari R. Mase, Giovanni Battista Migliori, Giovanni Sotgiu, Graham H. Bothamley, Jan L. Brozek, Adithya Cattamanchi, J. Peter Cegielski, Lisa Chen, Charles L. Daley, Tracy L. Dalton, Raquel Duarte, Federica Fregonese, C. Robert Horsburgh, Jr., Faiz Ahmad Khan, Fayez Kheir, Zhiyi Lan, Alfred Lardizabal, Michael Lauzardo, Joan M. Mangan, Suzanne M. Marks, Lindsay McKenna, Dick Menzies, Carole D. Mitnick, Diana M. Nilsen, Farah Parvez, Charles A. Peloquin, Ann Raftery, H. Simon Schaaf, Neha S. Shah, Jeffrey R. Starke, John W. Wilson, Jonathan M. Wortham, Terence Chorba, and Barbara Seaworth; on behalf of the American Thoracic Society, U.S. Centers for Disease Control and Prevention, European Respiratory Society, and Infectious Diseases Society of America THIS OFFICIAL CLINICAL PRACTICE GUIDELINE WAS APPROVED BY THE AMERICAN THORACIC SOCIETY, THE EUROPEAN RESPIRATORY SOCIETY, AND THE INFECTIOUS DISEASES SOCIETY OF AMERICA SEPTEMBER 2019, AND WAS CLEARED BY THE U.S. CENTERS FOR DISEASE CONTROL AND PREVENTION SEPTEMBER 2019 Background: The American Thoracic Society, U.S. Centers for was judged to be very low, because the data came Disease Control and Prevention, European Respiratory Society, and from observational studies with significant loss to follow-up Infectious Diseases Society of America jointly sponsored this new and imbalance in background regimens between comparator practice guideline on the treatment of drug-resistant tuberculosis groups. Good practices in the management of MDR-TB are (DR-TB). The document includes recommendations on the described. On the basis of the evidence review, a clinical strategy treatment of multidrug-resistant TB (MDR-TB) as well as tool for building a treatment regimen for MDR-TB is also isoniazid-resistant but rifampin-susceptible TB. -
Pretomanid Reference Number: CP.PMN.222 Effective Date: 03.01.20 Last Review Date: 02.20 Line of Business: Commercial, HIM, Medicaid Revision Log
Clinical Policy: Pretomanid Reference Number: CP.PMN.222 Effective Date: 03.01.20 Last Review Date: 02.20 Line of Business: Commercial, HIM, Medicaid Revision Log See Important Reminder at the end of this policy for important regulatory and legal information. Description Pretomanid is a nitroimidazooxazine antimycobacterial drug. FDA Approved Indication(s) Pretomanid is indicated as part of a combination regimen with bedaquiline and linezolid for the treatment of adults with pulmonary extensively drug resistant (XDR), treatment-intolerant or nonresponsive multidrug-resistant (MDR) tuberculosis (TB). Approval of this indication is based on limited clinical safety and efficacy data. This drug is indicated for use in a limited and specific population of patients. Limitation(s) of use: • Pretomanid tablets are not indicated for patients with: o Drug-sensitive (DS) tuberculosis o Latent infection due to Mycobacterium tuberculosis o Extra-pulmonary infection due to Mycobacterium tuberculosis o MDR-TB that is not treatment-intolerant or nonresponsive to standard therapy • Safety and effectiveness of pretomanid tablets have not been established for its use in combination with drugs other than bedaquiline and linezolid as part of the recommended dosing regimen. Policy/Criteria Provider must submit documentation (such as office chart notes, lab results or other clinical information) supporting that member has met all approval criteria. It is the policy of health plans affiliated with Centene Corporation® that pretomanid is medically necessary when the following criteria are met: I. Initial Approval Criteria A. Multi-Drug Resistant Tuberculosis (must meet all): 1. Diagnosis of pulmonary MDR-TB or XDR-TB; 2. Prescribed by or in consultation with an expert in the treatment of tuberculosis; 3. -
Model-Informed Drug Discovery and Development Strategy for the Rapid Development of Anti-Tuberculosis Drug Combinations
applied sciences Review Model-Informed Drug Discovery and Development Strategy for the Rapid Development of Anti-Tuberculosis Drug Combinations Rob C. van Wijk 1 , Rami Ayoun Alsoud 1, Hans Lennernäs 2 and Ulrika S. H. Simonsson 1,* 1 Department of Pharmaceutical Biosciences, Uppsala University, Uppsala 75123, Sweden; [email protected] (R.C.v.W.); [email protected] (R.A.A.) 2 Department of Pharmacy, Uppsala University, Uppsala 75123, Sweden; [email protected] * Correspondence: [email protected]; Tel.: +46-184-714-000 Received: 29 February 2020; Accepted: 25 March 2020; Published: 31 March 2020 Featured Application: Model-informed drug discovery and development (MID3) is proposed to be applied throughout the preclinical to clinical phases to provide an informative prediction of drug exposure and efficacy in humans in order to select novel anti-tuberculosis drug combinations for the treatment of tuberculosis. Abstract: The increasing emergence of drug-resistant tuberculosis requires new effective and safe drug regimens. However, drug discovery and development are challenging, lengthy and costly. The framework of model-informed drug discovery and development (MID3) is proposed to be applied throughout the preclinical to clinical phases to provide an informative prediction of drug exposure and efficacy in humans in order to select novel anti-tuberculosis drug combinations. The MID3 includes pharmacokinetic-pharmacodynamic and quantitative systems pharmacology models, machine learning and artificial intelligence, which integrates all the available knowledge related to disease and the compounds. A translational in vitro-in vivo link throughout modeling and simulation is crucial to optimize the selection of regimens with the highest probability of receiving approval from regulatory authorities. -
Treatment Outcomes of Adjunctive Surgery in Multidrug-Resistant And
MOJ Surgery Opinion Open Access Treatment outcomes of adjunctive surgery in multidrug-resistant and extensively drug-resistant tuberculosis Opinion Volume 5 Issue 2 - 2017 In 2013, the World Health Organization (WHO) reported that 3.5% and 20.5% of new and previously treated tuberculosis (TB) Attapon Cheepsattayakorn,1,2 Ruangrong cases were multidrug-resistant tuberculosis (MDR-TB, resistant 3 to both isoniazid and rifampicin), respectively, and 9.0% of them Cheepsattayakorn 1 developed extensively drug-resistant tuberculosis (XDR-TB, resistant 10th Zonal Tuberculosis and Chest Disease Center, Chiang Mai, Thailand to isoniazid, rifampicin, a fluoroquinolone, and 1 or greater injectable 2Department of Disease Control, Ministry of Public Health, agent). The WHO has estimated a global prevalence of 660,000 cases Thailand of MDR-TB and 150,000 MDR-TB related deaths annually. A previous 3Department of Pathology, Faculty of Medicine, Chiang Mai multi-country study revealed that among 1,278 MDR-TB cases, University, Thailand around 7% had XDR-TB. Only 136,000 MDR-TB cases (45.3%) Correspondence: Attapon Cheepsattayakorn, 10th Zonal among estimated 300,000 MDR-TB cases have been diagnosed and Tuberculosis and Chest Disease Center, 143 Sridornchai Road 97,000 cases (32.3%) are treated using appropriate regimens based on Changklan Muang Chiang Mai 50100 Thailand , Tel 66-531-407- drug susceptibility testing (DST). In 2013, the treatment success rate 67, 66 5-327-636-4, Fax 665-314-077-3, 665-327-359-0, of MDR-TB is only 48.0%; around 47,000 cases improved clinically Email [email protected]; [email protected] and biologically. -
Federal Register / Vol. 60, No. 80 / Wednesday, April 26, 1995 / Notices DIX to the HTSUS—Continued
20558 Federal Register / Vol. 60, No. 80 / Wednesday, April 26, 1995 / Notices DEPARMENT OF THE TREASURY Services, U.S. Customs Service, 1301 TABLE 1.ÐPHARMACEUTICAL APPEN- Constitution Avenue NW, Washington, DIX TO THE HTSUSÐContinued Customs Service D.C. 20229 at (202) 927±1060. CAS No. Pharmaceutical [T.D. 95±33] Dated: April 14, 1995. 52±78±8 ..................... NORETHANDROLONE. A. W. Tennant, 52±86±8 ..................... HALOPERIDOL. Pharmaceutical Tables 1 and 3 of the Director, Office of Laboratories and Scientific 52±88±0 ..................... ATROPINE METHONITRATE. HTSUS 52±90±4 ..................... CYSTEINE. Services. 53±03±2 ..................... PREDNISONE. 53±06±5 ..................... CORTISONE. AGENCY: Customs Service, Department TABLE 1.ÐPHARMACEUTICAL 53±10±1 ..................... HYDROXYDIONE SODIUM SUCCI- of the Treasury. NATE. APPENDIX TO THE HTSUS 53±16±7 ..................... ESTRONE. ACTION: Listing of the products found in 53±18±9 ..................... BIETASERPINE. Table 1 and Table 3 of the CAS No. Pharmaceutical 53±19±0 ..................... MITOTANE. 53±31±6 ..................... MEDIBAZINE. Pharmaceutical Appendix to the N/A ............................. ACTAGARDIN. 53±33±8 ..................... PARAMETHASONE. Harmonized Tariff Schedule of the N/A ............................. ARDACIN. 53±34±9 ..................... FLUPREDNISOLONE. N/A ............................. BICIROMAB. 53±39±4 ..................... OXANDROLONE. United States of America in Chemical N/A ............................. CELUCLORAL. 53±43±0 -
Appraisal of NICE's Cost-Effectiveness Thresholds for Assessing Orphan
Master's Degree Dissertation Appraisal of NICE’s cost-effectiveness thresholds for assessing orphan drugs Author: Ruodan Nan Master's Degree in Health Economics and Pharmacoeconomics UPF Barcelona School of Management Academic Year 2018 – 2021 Mentor: Laura Vallejo 1 Project performed within the framework of the Health Economics and Pharmacoeconomics program taught by Barcelona School of Management, a centre associated with Pompeu Fabra University 2 Abstract A rare disease is one that affects less than 1 in 2,000 in general population, and they are often chronic and life-threatening [1]. Due to the high cost to develop and small market for orphan drugs, historically manufacturers are reluctantly to explore the rare disease therapeutic areas. There is highly unmet need from patients with rare diseases. As the pharmaceutical expenditure rises, governments around the world are increasingly under pressure to contain the costs on drug reimbursement. Meanwhile patients with rare diseases need to be ensured for access to orphan drugs, and the industry needs to be incentivized to innovate. Health technology assessment and reimbursement bodies set out measures and policies for orphan drugs to be given market access. In this literature review, the landscape of health technology appraisals for orphan drugs in England UK is studied. The process for an orphan drug to gain marketing authorisation in the European Union (EU), the methods and criteria that used by National Institute for Health and Care Excellence (NICE) are reviewed. All orphan drugs with marketing authorisations from European Medicine Agency (EMA) are searched from the agency’s online database, whereas each of the authorised orphan drug is searched in NICE’s database for any technology appraisals and recommendations made for the orphan drug. -
Wo 2008/127291 A2
(12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date PCT (10) International Publication Number 23 October 2008 (23.10.2008) WO 2008/127291 A2 (51) International Patent Classification: Jeffrey, J. [US/US]; 106 Glenview Drive, Los Alamos, GOlN 33/53 (2006.01) GOlN 33/68 (2006.01) NM 87544 (US). HARRIS, Michael, N. [US/US]; 295 GOlN 21/76 (2006.01) GOlN 23/223 (2006.01) Kilby Avenue, Los Alamos, NM 87544 (US). BURRELL, Anthony, K. [NZ/US]; 2431 Canyon Glen, Los Alamos, (21) International Application Number: NM 87544 (US). PCT/US2007/021888 (74) Agents: COTTRELL, Bruce, H. et al.; Los Alamos (22) International Filing Date: 10 October 2007 (10.10.2007) National Laboratory, LGTP, MS A187, Los Alamos, NM 87545 (US). (25) Filing Language: English (81) Designated States (unless otherwise indicated, for every (26) Publication Language: English kind of national protection available): AE, AG, AL, AM, AT,AU, AZ, BA, BB, BG, BH, BR, BW, BY,BZ, CA, CH, (30) Priority Data: CN, CO, CR, CU, CZ, DE, DK, DM, DO, DZ, EC, EE, EG, 60/850,594 10 October 2006 (10.10.2006) US ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IS, JP, KE, KG, KM, KN, KP, KR, KZ, LA, LC, LK, (71) Applicants (for all designated States except US): LOS LR, LS, LT, LU, LY,MA, MD, ME, MG, MK, MN, MW, ALAMOS NATIONAL SECURITY,LLC [US/US]; Los MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PG, PH, PL, Alamos National Laboratory, Lc/ip, Ms A187, Los Alamos, PT, RO, RS, RU, SC, SD, SE, SG, SK, SL, SM, SV, SY, NM 87545 (US). -
ANTIBIOTIC INHIBITION of CATALYTIC RNA FUNCTION by Jeff
ANTIBIOTIC INHIBITION OF CATALYTIC RNA FUNCTION by Jeff Rogers B.Sc. (Honours), University of Regina, 1991 A THESIS SUBMITTED IN PARTIAL FULFILLMENT OF THE REQUIREMENTS FOR THE DEGREE OF DOCTOR OF PHILOSOPHY in THE FACULTY OF GRADUATE STUDIES (DEPARTMENT OF MICROBIOLOGY AND IMMUNOLOGY) We accept this thesis as conforming to the required standard THE UNIVERSITY OF BRITISH COLUMBIA August 1996 ©Jeff Rogers, 1996 In presenting this thesis in partial fulfilment of the requirements for an advanced degree at the University of British Columbia, I agree that the Library shall make it freely available for reference and study. I further agree that permission for extensive copying of this thesis for scholarly purposes may be granted by the head of my department or by his or her representatives. It is understood that copying or publication of this thesis for financial gain shall not be allowed without my written permission. Department The University of British Columbia Vancouver, Canada DE-6 (2/88) ii ABSTRACT A number of compounds inhibit group I intron splicing. Competitive inhibitors include deoxyguanosine, dideoxyguanosine, arginine and streptomycin; the non-competitive inhibitors include members of the aminoglycoside family of antibiotics. Further screening of a collection of antibiotics for their ability to inhibit group I intron splicing identified several novel compounds. In particular, the pseudodisaccharide antibiotic lysinomicin, the peptide antibiotics netropsin and distamycin, and the tetracycline analog chelocardin, were found to inhibit group I intron splicing at concentrations of 250 pM or lower. Inhibition of group I intron splicing by pseudodisaccharide antibiotics was studied in detail. Lysinomicin and three closely related compounds were found to inhibit the self splicing reaction of the Tetrahymena, Bacillus phage SP01 and T4 phage td and sun 7 group I introns at concentrations less than 50 u.M. -
Molecular Mechanism of Viomycin Inhibition of Peptide Elongation in Bacteria
Molecular mechanism of viomycin inhibition of peptide elongation in bacteria Mikael Holma, Anneli Borga, Måns Ehrenberga, and Suparna Sanyala,1 aDepartment of Cell and Molecular biology, Uppsala University, 75124 Uppsala, Sweden Edited by Joseph D. Puglisi, Stanford University School of Medicine, Stanford, CA, and approved December 16, 2015 (received for review September 2, 2015) Viomycin is a tuberactinomycin antibiotic essential for treating multi- activity of viomycin in genetic code translation (6) and its stabilization drug-resistant tuberculosis. It inhibits bacterial protein synthesis by of peptidyl tRNA in the A site (24). blocking elongation factor G (EF-G) catalyzed translocation of messen- In this study we used rapid kinetics methods in a state-of-the-art ger RNA on the ribosome. Here we have clarified the molecular aspects in vitro translation system with in vivo-like rates (25, 26) to charac- of viomycin inhibition of the elongating ribosome using pre-steady- terize the mechanism of viomycin inhibition of the peptide elongation state kinetics. We found that the probability of ribosome inhibition by cycle. We constructed a quantitative model for viomycin action with viomycin depends on competition between viomycin and EF-G for precise estimates of the model parameters. This model quantifies the binding to the pretranslocation ribosome, and that stable viomycin functional aspects of viomycin inhibition of translocation. It paves the binding requires an A-site bound tRNA. Once bound, viomycin stalls the way for deeper understanding of the basis of the antimicrobial activity ribosome in a pretranslocation state for a minimum of ∼45 s. This of viomycin and the evolution of viomycin resistance, thereby stalling time increases linearly with viomycin concentration. -
Stembook 2018.Pdf
The use of stems in the selection of International Nonproprietary Names (INN) for pharmaceutical substances FORMER DOCUMENT NUMBER: WHO/PHARM S/NOM 15 WHO/EMP/RHT/TSN/2018.1 © World Health Organization 2018 Some rights reserved. This work is available under the Creative Commons Attribution-NonCommercial-ShareAlike 3.0 IGO licence (CC BY-NC-SA 3.0 IGO; https://creativecommons.org/licenses/by-nc-sa/3.0/igo). Under the terms of this licence, you may copy, redistribute and adapt the work for non-commercial purposes, provided the work is appropriately cited, as indicated below. In any use of this work, there should be no suggestion that WHO endorses any specific organization, products or services. The use of the WHO logo is not permitted. If you adapt the work, then you must license your work under the same or equivalent Creative Commons licence. If you create a translation of this work, you should add the following disclaimer along with the suggested citation: “This translation was not created by the World Health Organization (WHO). WHO is not responsible for the content or accuracy of this translation. The original English edition shall be the binding and authentic edition”. Any mediation relating to disputes arising under the licence shall be conducted in accordance with the mediation rules of the World Intellectual Property Organization. Suggested citation. The use of stems in the selection of International Nonproprietary Names (INN) for pharmaceutical substances. Geneva: World Health Organization; 2018 (WHO/EMP/RHT/TSN/2018.1). Licence: CC BY-NC-SA 3.0 IGO. Cataloguing-in-Publication (CIP) data.