DOCSLIB.ORG

Human Medicines Highlights 2020 Authorisation of New Medicines

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text
Human Medicines Highlights 2020 Authorisation of New Medicines HUMAN MEDICINES HIGHLIGHTS 2020 AUTHORISATION OF NEW MEDICINES Key figures on the European Medicines Agency’s (EMA) recommendations for the authorisation of new medicines in 2020: POSITIVE NEW ACTIVE NEGATIVE WITHDRAWN OPINIONS SUBSTANCES OPINIONS APPLICATIONS 97 39 2 16 ADVANCED APPROVALS THERAPY CONDITIONAL UNDER MEDICINAL ORPHAN ACCELERATED MARKETING EXCEPTIONAL PRIME PRODUCTS MEDICINES* ASSESSMENTS AUTHORISATIONS CIRCUNSTANCES BIOSIMILARS 8 3 22 6 13 5 12 See more on the new recommendations from page 3. KEEPING PATIENTS SAFE Once a medicine has been put on the market, EMA and the European Union Member States continuously monitor its quality and benefit-risk balance. Important new safety advice issued in 2020 included: Recommendation to restrict the use Recommendation to update the safety of cyproterone acetate due to the information for hormone replacement risk of meningioma (brain tumor). therapy (HRT) used to treat symptoms The risk of meningioma increases with of the menopause. The updates are increasing cumulative doses. The use of based on evidence from a large study cyproterone acetate is contraindicated in published in The Lancet in August 2019, patients with a meningioma or a history which confirmed the known higher risk of meningioma. of breast cancer in women using HRT. The results showed that the risk may continue to be increased for ten years or Recommendation to update the more after stopping HRT, if it has been product information of fluroquinolone used for more than five years. antibiotics to reflect that systemic and inhaled fluroquinolones may increase the risk of heart valve regurgitation/ incompetence. See more on monitoring of medicines from page 12. These figures reflect EMA’s recommendations which are sent to the European Commission for the adoption of an EU-wide marketing authorisation. *This figure refers to medicines that had their orphan designation confirmedy b 31 December 2020. At the time of approval, orphan designations are reviewed by EMA’s Committee for Orphan Medicinal Products (COMP) to determine whether the information available to date allows maintaining the medicine’s orphan status. 2 MEDICINES RECOMMENDED FOR APPROVAL Pneumology/ Haematology/ Allergology Haemostaseology Arikayce liposomal Cancer Adakveo Atectura Breezhaler Aybintio Arsenic trioxide medac Bemrist Breezhaler Ayvakyt Arsenic trioxide Mylan Budesonide/Formoterol Teva Pharma B.V. Blenrep Azacitidine betapharm Enerzair breezhaler Cabazitaxel Accord Azacitidine Mylan Gencebok Daurismo Calquence Kaftrio Enhertu Elzonris Palforzia Equidacent Inrebic Trepulmix Lenalidomide Mylan Lenalidomide Krka Trixeo Aerosphere Nubeqa Lenalidomide Krka d.d. Zimbus Breezhaler Nyvepria Lenalidomide Krka d.d. Novo mesto Onbevzi Lumoxiti Phelinun Reblozyl Phesgo Ruxience Piqray Retsevmo Rozlytrek Sarclisa Immunology/ Sunitinib Accord Rheumatology/ Tecartus Transplantation Tukysa Zercepac Endocrinology Idefirix Jyseleca Cinacalcet Accordpharma Livogiva Givlaari Nepexto Insulin aspart Sanofi Qutavina Kixelle Yuflyma Lyumjev Ogluo Rybelsus Infections Fetcroja Hepcludex Heplisav B Vaccines Obiltoxaximab SFL Fluad Tetra Dovprela Neurology MenQuadfi (previously Pretomanid FGK) Mvabea Fampridine Accord Rekambys Supemtek Fingolimod Accord Rukobia Vaxchora Fintepla Tigecycline Accord Zabdeno Vocabria Libmeldy Xenleta Zeposia Xofluza Zolgensma PRIME ATMP Orphan medicine Accelerated assessment Conditional marketing authorisation Approval under exceptional circumstances Biosimilar Medicines that contain a new active substance are highlighted in blue 3 Covid-19 Metabolism Cardiovascular Comirnaty Nilemdo Apixaban Accord Veklury Nustendi Leqvio Rivaroxaban Accord Diagnostic agents Uro-nephrology Methylthioninium chloride Cosmo Pain Oxlumo Zynrelef Sibnayal Exparel Psychiatry Ophthalmology Paliperidone Janssen-Cilag International Dermatology Roclanda Staquis PRIME ATMP Orphan medicine Accelerated assessment Conditional marketing authorisation Approval under exceptional circumstances Biosimilar Medicines that contain a new active substance are highlighted in blue 4 OUTSTANDING CONTRIBUTIONS TO PUBLIC HEALTH Authorisation of new medicines is essential to advancing public health as they bring new opportunities to treat certain diseases. Below is a selection of medicines approved in 2020 that represent significant progress in their therapeutic areas: Cancer Immunology/Rheumatology/ Transplantation Blenrep to treat adult patients with relapsed Idefirix and refractory multiple myeloma who the first treatment for adult patients no longer respond to treatment with an waiting for a kidney transplant, who immunomodulatory agent, a proteasome are highly sensitised against tissue inhibitor and a CD-38 monoclonal from the donor and who have a positive antibody. crossmatch test against an available kidney from a deceased donor. Rozlytrek for the treatment of patients whose solid Neurology tumours have a neurotrophic tyrosine receptor kinase gene fusion, or patients Libmeldy with ROS1-positive advanced non-small to treat metachromatic leukodystrophy cell lung cancer. (MLD), a rare inherited metabolic disease that affects the nervous system. Tecartus for the treatment of adult patients with Zolgensma a rare cancer of white blood cells called to treat babies and young children with mantle cell lymphoma (MCL) when spinal muscular atrophy, a rare and the symptoms or the disease come often fatal genetic disease that causes back (relapse) or when they are not muscle weakness and progressive loss of responding (refractory) after two or more movement. lines of systemic therapy. Pneumology/Allergology Endocrinology Enerzair breezhaler Givlaari the first asthma triple combination the first treatment for acute hepatic therapy that includes an optional porphyria in adults and adolescents aged electronic sensor to collect data on the 12 years and older. use of the inhaler by the patient. Rybelsus Kaftrio for the treatment of adults with the first triple combination therapy insufficiently controlled type 2 diabetes for the treatment of cystic fibrosis in to improve glycaemic control as an patients aged 12 years and older who are adjunct to diet and exercise. It is the homozygous for the F508del mutation first glucagon-like peptide (GLP-1) in the cystic fibrosis transmembrane receptor agonist treatment - a class of conductance regulator (CFTR) gene or non-insulin medicines for people with heterozygous for F508del in the CFTR type 2 diabetes - developed for oral use, gene with a minimal function (MF) providing patients with another option to mutation. treat the disease without injections. Uro-nephrology Infections Oxlumo Rekambys and Vocabria for the treatment of the rare inherited to be used together for the treatment of disorder primary hyperoxaluria type 1. patients with human immunodeficiency virus type 1 (HIV-1) infection. The two Vaccines medicines are the first ARVs that come in Zabdeno and Mvabea a long-acting injectable formulation. the two components of a new vaccine that provides active immunisation to prevent Ebola virus disease in individuals 5 aged one year and older. Highlights on COVID-19 EMA is contributing to tackling the pneumonia who require supplemental COVID-19 pandemic by expediting the oxygen. In December, the Committee development and approval of safe and recommended a change to the product effective treatments and vaccines while information for Veklury to provide clearer maintaining its high standards of quality, instructions in which COVID-19 patients safety and efficacy. This includes the requiring supplementary oxygen it should reduction of timelines for evaluation and be used. the use of rolling reviews. The Agency also In September 2020, the Committee provides reliable information to patients and completed its review of results from healthcare professionals. the RECOVERY study arm that involved In December 2020, EMA’s human medicines the use of the corticosteroid medicine committee (CHMP) recommended granting dexamethasone in the treatment of patients a conditional marketing authorisation with COVID-19 admitted to hospital, for the vaccine Comirnaty, developed by and concluded that dexamethasone can BioNTech and Pfizer, to prevent coronavirus be considered a treatment option for disease 2019 (COVID-19) in people from 16 patients who require oxygen therapy years of age. (from supplemental oxygen to mechanical By the end of the year, one vaccine ventilation). (Moderna’s mRNA-1273 COVID-19 vaccine) Throughout the year, EMA issued reminders was under evaluation by the Committee to healthcare professionals to closely and rolling reviews of two additional monitor patients with COVID-19 who vaccines were also ongoing – one for the received chloroquine or hydroxychloroquine, vaccine being developed by AstraZeneca given the serious side effects that can result with the University of Oxford and one for from treatment with these medicines. Both the vaccine from Janssen Cilag. chloroquine and hydroxychloroquine, which In June 2020, the CHMP recommended are authorised for malaria and certain granting a conditional marketing autoimmune diseases, have been used authorisation to Veklury (remdesivir) for to treat patients with COVID-19 but their the treatment of COVID-19 in adults and beneficial effects in this patient population adolescents from 12 years of age with are not established. 6 11th medicine recommended for use outside the European Union In July 2020, EMA’s human medicines This is the eleventh medicine recommended by committee (CHMP) adopted a positive EMA
Load more

Recommended publications
  • 996.Full-Text.Pdf
    Published OnlineFirst April 6, 2010; DOI: 10.1158/1535-7163.MCT-09-0960 Research Article Molecular Cancer Therapeutics Potent Preclinical Impact of Metronomic Low-Dose Oral Topotecan Combined with the Antiangiogenic Drug Pazopanib for the Treatment of Ovarian Cancer Kae Hashimoto1, Shan Man1, Ping Xu1, William Cruz-Munoz1, Terence Tang1, Rakesh Kumar3, and Robert S. Kerbel1,2 Abstract Low-dose metronomic chemotherapy has shown promising activity in many preclinical and some phase II clinical studies involving various tumor types. To evaluate further the potential therapeutic impact of met- ronomic chemotherapy for ovarian cancer, we developed a preclinical model of advanced human ovarian cancer and tested various low-dose metronomic chemotherapy regimens alone or in concurrent combination with an antiangiogenic drug, pazopanib. Clones of the SKOV-3 human ovarian carcinoma cell line expressing a secretable β-subunit of human choriogonadotropic (β-hCG) protein and firefly luciferase were generated and evaluated for growth after orthotopic (i.p.) injection into severe combined immunodeficient mice; a high- ly aggressive clone, SKOV-3-13, was selected for further study. Mice were treated beginning 10 to 14 days after injection of cells when evidence of carcinomatosis-like disease in the peritoneum was established as assessed by imaging analysis. Chemotherapy drugs tested for initial experiments included oral cyclophos- phamide, injected irinotecan or paclitaxel alone or in doublet combinations with cyclophosphamide; the re- sults indicated that metronomic cyclophosphamide had no antitumor activity whereas metronomic irinotecan had potent activity. We therefore tested an oral topoisomerase-1 inhibitor, oral topotecan, at optimal biological dose of 1 mg/kg/d. Metronomic oral topotecan showed excellent antitumor activity, the extent of which was significantly enhanced by concurrent pazopanib, which itself had only modest activity, with 100% survival values of the drug combination after six months of continuous therapy.
    [Show full text]
  • Assessment of Overall Survival, Quality of Life, And
    Supplementary Online Content Salas-Vega S, Iliopoulos O, Mossialos E. Assesment of overall survival, quality of life, and safety benefits associated with new cancer medicines [published online December 29, 2016]. JAMA Oncol. doi:10.1001/jamaoncol.2016.4166 eTable 1. Therapeutic profile of all cancer medicines approved by the FDA between 2003- 2013 eTable 2. Regulatory evidence in support of classification of drug clinical benefits eFigure 1. Number of cancer drugs that were evaluated by all three HTA agencies, sorted by magnitude of clinical benefits eTable 3. Interagency agreement–Krippendorff’s alpha coefficients eMethods This supplementary material has been provided by the authors to give readers additional information about their work. Online-Only Supplement © 2016 American Medical Association. All rights reserved. Downloaded From: https://jamanetwork.com/ on 09/25/2021 Clinical value of cancer medicines Contents eExhibits ......................................................................................................................................................... 3 eTable 1. Therapeutic profile of all cancer medicines approved by the FDA between 2003- 2013 (Summary of eTable 2) ................................................................................................................... 3 eTable 2. Regulatory evidence in support of classification of drug clinical benefits ....................... 6 eFigure 1. Number of cancer drugs that were evaluated by all three HTA agencies, sorted by magnitude of clinical benefits
    [Show full text]
  • Where Do Novel Drugs of 2016 Fit In?
    FORMULARY JEOPARDY: WHERE DO NOVEL DRUGS OF 2016 FIT IN? Maabo Kludze, PharmD, MBA, CDE, BCPS, Associate Director Elizabeth A. Shlom, PharmD, BCPS, SVP & Director Clinical Pharmacy Program Acurity, Inc. Privileged and Confidential August 15, 2017 Privileged and Confidential Program Objectives By the end of the presentation, the pharmacist or pharmacy technician participant will be able to: ◆ Identify orphan drugs and first-in-class medications approved by the FDA in 2016. ◆ Describe the role of new agents approved for use in oncology patients. ◆ Identify and discuss the role of novel monoclonal antibodies. ◆ Discuss at least two new medications that address public health concerns. Neither Dr. Kludze nor Dr. Shlom have any conflicts of interest in regards to this presentation. Privileged and Confidential 2016 NDA Approvals (NMEs/BLAs) ◆ Nuplazid (primavanserin) P ◆ Adlyxin (lixisenatide) ◆ Ocaliva (obeticholic acid) P, O ◆ Anthim (obitoxaximab) O ◆ Rubraca (rucaparib camsylate) P, O ◆ Axumin (fluciclovive F18) P ◆ Spinraza (nusinersen sodium) P, O ◆ Briviact (brivaracetam) ◆ Taltz (ixekizumab) ◆ Cinqair (reslizumab) ◆ Tecentriq (atezolizumab) P ◆ Defitelio (defibrotide sodium) P, O ◆ Venclexta (venetoclax) P, O ◆ Epclusa (sofosburvir and velpatasvir) P ◆ Xiidra (lifitigrast) P ◆ Eucrisa (crisaborole) ◆ Zepatier (elbasvir and grazoprevir) P ◆ Exondys 51 (eteplirsen) P, O ◆ Zinbyrta (daclizumab) ◆ Lartruvo (olaratumab) P, O ◆ Zinplava (bezlotoxumab) P ◆ NETSTPOT (gallium Ga 68 dotatate) P, O O = Orphan; P = Priority Review; Red = BLA Privileged and Confidential History of FDA Approvals Privileged and Confidential Orphan Drugs ◆FDA Office of Orphan Products Development • Orphan Drug Act (1983) – drugs and biologics . “intended for safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S.
    [Show full text]
  • A Phase II Study of Adjuvant Chemotherapy of Tegafur–Uracil For
    ANTICANCER RESEARCH 36 : 6505-6510 (2016) doi:10.21873/anticanres.11250 A Phase II Study of Adjuvant Chemotherapy of Tegafur –Uracil for Patients with Breast Cancer with HER2-negative Pathologic Residual Invasive Disease After Neoadjuvant Chemotherapy SATORU TANAKA 1, MITSUHIKO IWAMOTO 2, KOSEI KIMURA 2, YUKO TAKAHASHI 3, HIYOYA FUJIOKA 2, NAYUKO SATO 4, RISA TERASAWA 2, KANAKO KAWAGUCHI 2, AYANA IKARI 2, TOMO TOMINAGA 2, SAKI MAEZAWA 2, NODOKA UMEZAKI 2, JUNNA MATSUDA 2 and KAZUHISA UCHIYAMA 2 1Breast Surgery, National Hospital Organization Osaka-Minami Medical Center, Kawachinagano, Japan; 2Breast and Endocrine Surgery, Osaka Medical College Hospital, Takatsuki, Japan; 3Breast and Endocrine Surgery, Hirakata City Hospital, Hirakata, Japan; 4Breast Surgery, Saiseikai Suita Hospital, Suita, Japan Abstract. Background: There is no consensus on the need an opportunity for down-staging to avoid mastectomy for for adjuvant chemotherapy for patients with pathological inoperable tumors or breast-conserving surgery in cases residual invasive breast cancer (non-pCR) after neoadjuvant originally requiring mastectomy (1, 2). Anthracyclines and chemotherapy (NAC). We evaluated the tolerability and taxanes have been used as the standard chemotherapeutic safety of tegafur-uracil (UFT) as adjuvant chemotherapy for regimens for breast cancer, however, tumoral estrogen patients with human epidermal growth factor receptor 2- receptor (ER) and human epidermal growth factor receptor negative breast cancer that resulted in non-pCR after NAC. 2 (HER2) statuses are important predictors of response to Patients and Methods: We treated patients with 270 mg/m 2 chemotherapy (3-5). Sensitivity to NAC is lower in ER- UFT per day for 2 years after definitive surgery and positive and HER2-negative cancer than in other subtypes.
    [Show full text]
  • Adjuvant Tegafur-Uracil (UFT)
    Yen et al. World Journal of Surgical Oncology (2021) 19:124 https://doi.org/10.1186/s12957-021-02233-2 RESEARCH Open Access Adjuvant tegafur-uracil (UFT) or S-1 monotherapy for advanced gastric cancer: a single center experience Hung-Hsuan Yen1,2, Chiung-Nien Chen2, Chi-Chuan Yeh2,3 and I-Rue Lai2,4* Abstract Background: Adjuvant tegafur-gimeracil-oteracil (S-1) is commonly used for gastric cancer in Asia, and tegafur- uracil (UFT) is another oral fluoropyrimidine when S-1 is unavailable. The real-world data of adjuvant UFT has less been investigated. Methods: Patients with pathological stage II-IIIB (except T1) gastric cancer receiving adjuvant UFT or S-1 monotherapy after D2 gastrectomy were included. Usage of UFT or S-1 was based on reimbursement policy of the Taiwanese healthcare system. The characteristics, chemotherapy completion rates, and 5-year recurrence-free survival (RFS) and overall survival (OS), were compared between these two groups. Results: From 2005 to 2016, 86 eligible patients were included. Most tumor characteristics were similar between the UFT group (n = 37; age 59.1 ± 13.9 years) and S-1 group (n = 49; age 56.3 ± 10.7 years), except there were significantly more Borrmann type III/IV (86.5% versus 67.3%; p = 0.047) and T4 (56.8% versus 10.2%; p < 0.001) lesions in the UFT group than in the S-1 group. The chemotherapy complete rates were similar in the two groups. The 5-year RFS was 56.1% in the UFT group and 59.6% in the S-1 group (p = 0.71), and the 5-year OS was 78.3% in the UFT group and 73.1% in the S-1 group (p = 0.48).
    [Show full text]
  • Postsurgical Oral Administration of Uracil and Tegafur Inhibits Progression of Micrometastasis of Human Breast Cancer Cells in Nude Mice1
    Vol. 3, 653-659, May 1997 Clinical Cancer Research 653 Postsurgical Oral Administration of Uracil and Tegafur Inhibits Progression of Micrometastasis of Human Breast Cancer Cells in Nude Mice1 Junichi Kurebayashi,2 Mamoru NUkatSUka, inhibited by the administration of either dose of UFT. In Akio Fujioka, Hitoshi Saito, Setsuo Takeda, conclusion, this new pOStSUrgIcal metastasis model may be useful for evaluating the efficacy of agents used in the post- Norio Unemi, Hiroyuki Fukumori, operative adjuvant setting. UFI may be an effective drug for MasafUmi Kurosumi, Hiroshi Sonoo, Inhibiting the progression of mlcrometastasis after surgery. and Robert B. Dickson Department of Breast and Thyroid Surgery, Kawasaki Medical School, 577 Matsushima, Kurashiki, Okayama 701-01, Japan [J. K., INTRODUCTION H. So.]; Taiho Pharmaceutical Co. Ltd., Tokyo 101, Japan EM. N., The efficacy of postoperative adjuvant chemotherapy A. F., H. Sa., S. T., N. U., H. F.]; Department of Pathology, Saitama and/or endocrine therapy in reducing the mortality of breast Cancer Center, Saitama 362, Japan [M. K.]; and Lombardi Cancer cancer patients has been widely accepted on the basis of the data Center, Georgetown University Medical Center, Washington, DC 20007 [R. B. D.] from many prospective clinical trials (1-3). These agentsused in the postoperative adjuvant setting have already been proven to be effective in the treatment of advanced breast cancer. The ABSTRACT antitumor activity of these agents is believed to be involved in We recently established a metastasis model In nude inhibition of the progression of micrometastasis, which may mice using the MKL-4 cell line, a contransfectant of the already exist at the time of surgery.
    [Show full text]
  • Guidance on the Use of Capecitabine and Tegafur with Uracil for Metastatic Colorectal Cancer
    Guidance on the use of capecitabine and tegafur with uracil for metastatic colorectal cancer Technology appraisal guidance Published: 27 May 2003 www.nice.org.uk/guidance/ta61 © NICE 2020. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights). Guidance on the use of capecitabine and tegafur with uracil for metastatic colorectal cancer (TA61) Your responsibility The recommendations in this guidance represent the view of NICE, arrived at after careful consideration of the evidence available. When exercising their judgement, health professionals are expected to take this guidance fully into account, alongside the individual needs, preferences and values of their patients. The application of the recommendations in this guidance are at the discretion of health professionals and their individual patients and do not override the responsibility of healthcare professionals to make decisions appropriate to the circumstances of the individual patient, in consultation with the patient and/or their carer or guardian. Commissioners and/or providers have a responsibility to provide the funding required to enable the guidance to be applied when individual health professionals and their patients wish to use it, in accordance with the NHS Constitution. They should do so in light of their duties to have due regard to the need to eliminate unlawful discrimination, to advance equality of opportunity and to reduce health inequalities. Commissioners and providers have a responsibility to promote an environmentally sustainable health and care system and should assess and reduce the environmental impact of implementing NICE recommendations wherever possible. © NICE 2020. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- Page 2 of conditions#notice-of-rights).
    [Show full text]
  • CDER Therapeutic Biologic Products List
    CDER Therapeutic Biologic Products This list is intended to include all the Center for Drug Evaluation and Research (CDER) user fee billable therapeutic biological products and potencies approved under Section 351 of the Public Health Service Act. The Orange Book includes a section entitled "Drug Products with Approval under Section 505 of the Act Administered by CBER." Included on that list are several products that have been transferred to CDER which would be considered billable also. Program fees are assessed for each potency in which the approved (non-revoked, non-suspended) product is manufactured in final dosage form. When evaluating the specific strength or potency of a drug in final dosage form for purposes of assessing program fees for liquid parenteral biological products, CDER intends to take into consideration both the total amount of drug substance in mass or units of activity in a product and the concentration of drug substance (mass or units of activity per unit volume of product). Biologic products considered to have a different strength or potency in a final dosage form will be given separate entries in the Biologics List and assessed separate program fees. An auto-injector that has the same strength or potency as a prefilled syringe or vial will generally be assessed a separate prescription drug program fee. In certain circumstances, products which have been discontinued from marketing but are still licensed are not assessed program fees. Those products are identified on the CDER Discontinued Biologic Product List section. The potency information contained in this list is based on information in our database.
    [Show full text]
  • The Clinical and Economic Benefits of Capecitabine and Tegafur with Uracil in Metastatic Colorectal Cancer
    British Journal of Cancer (2006) 95, 27 – 34 & 2006 Cancer Research UK All rights reserved 0007 – 0920/06 $30.00 www.bjcancer.com The clinical and economic benefits of capecitabine and tegafur with uracil in metastatic colorectal cancer *,1 1 1 2 3 4 SE Ward , E Kaltenthaler , J Cowan , M Marples , B Orr and MT Seymour 1School of Health and Related Research, University of Sheffield, Regent Court, 30 Regent Street, Sheffield S1 4DA, UK; 2Cancer Research Centre, Weston 3 4 Park Hospital, Sheffield S10 2SJ, UK; Weston Park Hospital, Sheffield S10 2SJ, UK; Cancer Research UK Centre, University of Leeds, Cookridge Hospital, Leeds LS16 6QB, UK Two oral fluoropyrimidine therapies have been introduced for metastatic colorectal cancer. One is a 5-fluorouracil pro-drug, Clinical Studies capecitabine; the other is a combination of tegafur and uracil administered together with leucovorin. The purpose of this study was to compare the clinical effectiveness and cost-effectiveness of these oral therapies against standard intravenous 5-fluorouracil regimens. A systematic literature review was conducted to assess the clinical effectiveness of the therapies and costs were calculated from the UK National Health Service perspective for drug acquisition, drug administration, and the treatment of adverse events. A cost- minimisation analysis was used; this assumes that the treatments are of equal efficacy, although direct randomised controlled trial (RCT) comparisons of the oral therapies with infusional 5-fluorouracil schedules were not available. The cost-minimisation analysis showed that treatment costs for a 12-week course of capecitabine (d2132) and tegafur with uracil (d3385) were lower than costs for the intravenous Mayo regimen (d3593) and infusional regimens on the de Gramont (d6255) and Modified de Gramont (d3485) schedules over the same treatment period.
    [Show full text]
  • Metronomic Oral Topotecan Prolongs Survival and Reduces Liver
    Gut Online First, published on April 28, 2012 as 10.1136/gutjnl-2011-301585 Colon cancer ORIGINAL ARTICLE Gut: first published as 10.1136/gutjnl-2011-301585 on 28 April 2012. Downloaded from Metronomic oral topotecan prolongs survival and reduces liver metastasis in improved preclinical orthotopic and adjuvant therapy colon cancer models Christina Hackl, Shan Man, Giulio Francia, Chloe Milsom, Ping Xu, Robert S Kerbel < Additional figures are ABSTRACT published online only. To view Objective Advanced and recurrent diseases are the Significance of this study these files please visit the major causes of death in colon cancer. No standard journal online (http://gut.bmj. com/content/early/recent). preclinical model addresses advanced disease and What is already known on this subject? spontaneous metastasis after orthotopic tumour growth. Department of Medical < Local recurrence and metastatic disease remain In this study, the authors report the establishment of Biophysics, Molecular and the leading causes of death in colon cancer. This such standardised orthotopic mouse models of colon Cellular Biology Research, is likely to be a major factor in the disparity of Sunnybrook Research Institute, cancer and their use in evaluating metronomic topotecan therapy results that are so frequently obtained University of Toronto, Toronto, alone or in combination with standard chemotherapy. using localised, ectopically implanted tumour Ontario, Canada Design Human colon cancer cell lines, transfected with models with those observed in clinical investi- human chorionic gonadotropin and luciferase, were Correspondence to gations, clearly highlighting the need for injected orthotopically into the caecal wall of severe Dr Christina Hackl, Molecular & improved preclinical models. Cellular Biology Research, combined immunodeficient mice, intrasplenically or < Metronomic cyclophosphamide plus uracil/ Sunnybrook Research Institute, subcutaneously.
    [Show full text]
  • Studies on Antitoxin Use for Postexposure Prophylaxis (PEP)
    ACIP Anthrax Vaccine Work Group William Bower, MD, FIDSA Epidemiology Team Lead Bacterial Special Pathogens October 23, 2018 National Center for Emerging and Zoonotic Disease Division of High-Consequence Pathogens and Pathology Outline Overview of anthrax antitoxins Studies on antitoxin use for postexposure prophylaxis (PEP) . Anthrax antitoxin for PEP and survival in animal models . Anthrax antitoxin effect on acquired immunity . Anthrax antitoxin use with anthrax vaccine adsorbed (AVA) Work group discussions Guidance on anthrax antitoxin use for PEP 2 Anthrax Pathogenesis 3 Background – Anthrax Antitoxins There are currently three FDA approved anthrax antitoxins RAXIBACUMAB . Initial U.S. Approval: 2012 . Human IgG1λ monoclonal antibody that binds the protective antigen (PA) component of Bacillus anthracis toxin ANTHRASIL (Anthrax Immune Globulin Intravenous (AIGIV)) . Initial U.S. Approval: 2015 . Purified human IgG containing polyclonal antibodies that bind the protective antigen (PA) component of B. anthracis lethal and edema toxins collected from individuals immunized with AVA ANTHIM (obiltoxaximab) . Initial U.S. Approval: 2016 . Chimeric IgG1 kappa monoclonal antibody that binds the PA component of B. anthracis toxin 4 Background – Anthrax Antitoxins Indications Anthrax antitoxins all have an indication for the treatment of adult and pediatric patients with inhalation anthrax due to B. anthracis in combination with appropriate antimicrobials The monoclonal antitoxins, raxibacumab and obiltoxaximab, are also indicated for
    [Show full text]
  • ENTRY WATCH 2016 Published by the Patented Medicine Prices Review Board June 2018 Meds Entry Watch, 2016 Is Available in Electronic Format on the PMPRB Website
    MEDS ENTRY WATCH 2016 Published by the Patented Medicine Prices Review Board June 2018 Meds Entry Watch, 2016 is available in electronic format on the PMPRB website. Une traduction de ce document est également disponible en français sous le titre : Veille des médicaments mis en marché, 2016 Patented Medicine Prices Review Board Standard Life Centre Box L40 333 Laurier Avenue West Suite 1400 Ottawa, ON K1P 1C1 Tel.: 1-877-861-2350 TTY 613-288-9654 Email: [email protected] Web: www.pmprb-cepmb.gc.ca ISSN 2560-6204 Cat. No.: H79-12E-PDF © Her Majesty the Queen in Right of Canada, as represented by the NPDUIS initiative of the Patented Medicine Prices Review Board, 2018 MEDS ENTRY WATCH 2016 About the PMPRB Acknowledgements The Patented Medicine Prices Review Board This report was prepared by the Patented (PMPRB) is a respected public agency that makes Medicine Prices Review Board (PMPRB) a unique and valued contribution to sustainable as part of the National Prescription Drug spending on pharmaceuticals in Canada by: Utilization Information System (NPDUIS). ~ providing stakeholders with price, cost and The PMPRB would like to acknowledge the utilization information to help them make timely contributions of and knowledgeable drug pricing, purchasing and ~ The members of the NPDUIS Advisory reimbursement decisions; and Committee for their expert oversight and ~ acting as an effective check on the patent rights guidance in the preparation of this report. of pharmaceutical manufacturers through the ~ PMPRB NPDUIS staff for their contribution responsible and efficient use of its consumer to the analytical content of the report: protection powers.
    [Show full text]