DOCSLIB.ORG

Minutes of the CHMP Meeting 25-29 January 2021

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text
Minutes of the CHMP Meeting 25-29 January 2021 04 March 2021 EMA/CHMP/128746/2021 Human Medicines Division Committee for medicinal products for human use (CHMP) Minutes for the meeting on 25-29 January 2021 Chair: Harald Enzmann – Vice-Chair: Bruno Sepodes Disclaimers Some of the information contained in this set of minutes is considered commercially confidential or sensitive and therefore not disclosed. With regard to intended therapeutic indications or procedure scopes listed against products, it must be noted that these may not reflect the full wording proposed by applicants and may also vary during the course of the review. Additional details on some of these procedures will be published in the CHMP meeting highlights once the procedures are finalised and start of referrals will also be available. Of note, these minutes are a working document primarily designed for CHMP members and the work the Committee undertakes. Note on access to documents Some documents mentioned in the minutes cannot be released at present following a request for access to documents within the framework of Regulation (EC) No 1049/2001 as they are subject to on-going procedures for which a final decision has not yet been adopted. They will become public when adopted or considered public according to the principles stated in the Agency policy on access to documents (EMA/127362/2006). Official address Domenico Scarlattilaan 6 ● 1083 HS Amsterdam ● The Netherlands Address for visits and deliveries Refer to www.ema.europa.eu/how-to-find-us Send us a question Go to www.ema.europa.eu/contact Telephone +31 (0)88 781 6000 An agency of the European Union © European Medicines Agency, 2021. Reproduction is authorised provided the source is acknowledged. Table of contents 1. Introduction 8 1.1. Welcome and declarations of interest of members, alternates and experts ............ 8 1.2. Adoption of agenda ................................................................................................ 8 1.3. Adoption of the minutes ......................................................................................... 8 2. Oral Explanations 9 2.1. Pre-authorisation procedure oral explanations ....................................................... 9 2.1.1. BroPair Spiromax - salmeterol xinafoate / fluticasone propionate - EMEA/H/C/005591 ....... 9 2.1.2. Seffalair Spiromax - salmeterol xinafoate / fluticasone propionate - EMEA/H/C/004881 ...... 9 2.1.3. COVID-19 vaccine AstraZeneca – COVID-19 Vaccine (ChAdOx1-S [recombinant]) – EMEA/H/C/005675 ..................................................................................................... 9 2.2. Re-examination procedure oral explanations ....................................................... 10 2.3. Post-authorisation procedure oral explanations ................................................... 10 2.3.1. Epidyolex - cannabidiol - Orphan - EMEA/H/C/004675/II/0005 ...................................... 10 2.3.2. Elebrato Ellipta - fluticasone furoate / umeclidinium / vilanterol - EMEA/H/C/004781/X/0014/G Temybric Ellipta - fluticasone furoate / umeclidinium / vilanterol - EMEA/H/C/005254/X/0004/G TrelegyEllipta - fluticasone furoate / umeclidinium / vilanterol - EMEA/H/C/004363/X/0012/G ............................................................................................................................. 10 2.4. Referral procedure oral explanations ................................................................... 11 3. Initial applications 11 3.1. Initial applications; Opinions ................................................................................ 11 3.1.1. Alymsys - bevacizumab - EMEA/H/C/005286 ............................................................... 11 3.1.2. BroPair Spiromax - salmeterol xinafoate / fluticasone propionate - EMEA/H/C/005591 ..... 11 3.1.3. Byfavo - remimazolam - EMEA/H/C/005246 ................................................................ 12 3.1.4. Kesimpta - ofatumumab - EMEA/H/C/005410 .............................................................. 12 3.1.5. Nexpovio - selinexor - Orphan - EMEA/H/C/005127 ..................................................... 13 3.1.6. Ontozry - cenobamate - EMEA/H/C/005377 ................................................................ 13 3.1.7. Oyavas - bevacizumab - EMEA/H/C/005556 ................................................................ 14 3.1.8. Pemazyre - pemigatinib - Orphan - EMEA/H/C/005266 ................................................. 14 3.1.9. Seffalair Spiromax - salmeterol xinafoate / fluticasone propionate - EMEA/H/C/004881 .... 15 3.1.10. Sogroya - somapacitan - Orphan - EMEA/H/C/005030 .................................................. 15 3.1.11. Thiotepa Riemser - thiotepa - EMEA/H/C/005434......................................................... 16 3.1.12. Vazkepa - icosapent ethyl - EMEA/H/C/005398 ........................................................... 16 3.1.13. COVID-19 vaccine AstraZeneca – COVID-19 Vaccine (ChAdOx1-S [recombinant]) – EMEA/H/C/005675 ................................................................................................... 17 3.1.14. Comirnaty - COVID-19 mRNA vaccine (nucleoside-modified) - EMEA/H/C/005735 ........... 17 3.1.15. COVID-19 Vaccine Moderna – COVID-19 mRNA vaccine (nucleoside-modified) - EMEA/H/C/005791 ................................................................................................... 18 Committee for medicinal products for human use (CHMP) EMA/CHMP/128746/2021 Page 2/68 3.2. Initial applications; List of outstanding issues (Day 180; Day 120 for procedures with accelerated assessment timetable) ...................................................................... 19 3.2.1. abiraterone acetate - EMEA/H/C/005408 .................................................................... 19 3.2.2. hydrocortisone - Orphan - EMEA/H/C/005105 ............................................................. 19 3.2.3. dostarlimab - EMEA/H/C/005204 ............................................................................... 19 3.2.4. estetrol / drospirenone - EMEA/H/C/005336................................................................ 19 3.2.5. risdiplam - Orphan - EMEA/H/C/005145 ..................................................................... 20 3.2.6. pralsetinib - EMEA/H/C/005413 ................................................................................. 20 3.2.7. estetrol / drospirenone - EMEA/H/C/005382................................................................ 20 3.2.8. ponesimod - EMEA/H/C/005163 ................................................................................ 20 3.2.9. tanezumab - EMEA/H/C/005189 ................................................................................ 21 3.2.10. relugolix / estradiol / norethisterone acetate - EMEA/H/C/005267 .................................. 21 3.3. Initial applications; List of questions (Day 120; Day 90 for procedures with accelerated assessment timetable) ...................................................................... 21 3.3.1. abrocitinib - EMEA/H/C/005452 ................................................................................. 21 3.3.2. artesunate - Orphan - EMEA/H/C/005550 ................................................................... 22 3.3.3. avalglucosidase alfa - Orphan - EMEA/H/C/005501 ...................................................... 22 3.3.4. ranibizumab - EMEA/H/C/005545 .............................................................................. 22 3.3.5. lonapegsomatropin - Orphan - EMEA/H/C/005367 ....................................................... 22 3.3.6. adalimumab - EMEA/H/C/005548 .............................................................................. 23 3.3.7. teriparatide - EMEA/H/C/004932 ............................................................................... 23 3.3.8. pegcetacoplan - Orphan - EMEA/H/C/005553 .............................................................. 23 3.3.9. ripretinib - Orphan - EMEA/H/C/005614 ..................................................................... 23 3.3.10. rivaroxaban - EMEA/H/C/005600 ............................................................................... 23 3.3.11. autologous glioma tumor cells, inactivated / autologous glioma tumor cell lysates, inactivated / allogeneic glioma tumor cells, inactivated / allogeneic glioma tumor cell lysates, inactivated - Orphan - ATMP - EMEA/H/C/003693 .......................................................................... 24 3.3.12. elivaldogene autotemcel - Orphan - ATMP - EMEA/H/C/003690 ..................................... 24 3.4. Update on on-going initial applications for Centralised procedure ........................ 24 3.4.1. zanubrutinib - Orphan - EMEA/H/C/004978 ................................................................. 24 3.4.2. leuprorelin - EMEA/H/C/005034 ................................................................................. 25 3.4.3. doxorubicin hydrochloride - EMEA/H/C/005330 ........................................................... 25 3.4.4. pitolisant - EMEA/H/C/005117 ................................................................................... 25 3.4.5. doxorubicin - EMEA/H/C/005320 ............................................................................... 26 3.4.6. sildenafil - EMEA/H/C/005439 ................................................................................... 26 3.4.7. sitagliptin - EMEA/H/C/005598 .................................................................................
Load more

Recommended publications
  • Endothelin System and Therapeutic Application of Endothelin Receptor
    xperim ACCESS Freely available online & E en OPEN l ta a l ic P in h l a C r m f o a c l a o n l o r g u y o J Journal of ISSN: 2161-1459 Clinical & Experimental Pharmacology Research Article Endothelin System and Therapeutic Application of Endothelin Receptor Antagonists Abebe Basazn Mekuria, Zemene Demelash Kifle*, Mohammedbrhan Abdelwuhab Department of Pharmacology, School of Pharmacy, College of Medicine and Health Sciences, University of Gondar, Gondar, Ethiopia ABSTRACT Endothelin is a 21 amino acid molecule endogenous potent vasoconstrictor peptide. Endothelin is synthesized in vascular endothelial and smooth muscle cells, as well as in neural, renal, pulmonic, and inflammatory cells. It acts through a seven transmembrane endothelin receptor A (ETA) and endothelin receptor B (ETB) receptors belongs to G protein-coupled rhodopsin-type receptor superfamily. This peptide involved in pathogenesis of cardiovascular disorder like (heart failure, arterial hypertension, myocardial infraction and atherosclerosis), renal failure, pulmonary arterial hypertension and it also involved in pathogenesis of cancer. Potentially endothelin receptor antagonist helps the treatment of the above disorder. Currently, there are a lot of trails both per-clinical and clinical on endothelin antagonist for various cardiovascular, pulmonary and cancer disorder. Some are approved by FAD for the treatment. These agents are including both selective and non-selective endothelin receptor antagonist (ETA/B). Currently, Bosentan, Ambrisentan, and Macitentan approved
    [Show full text]
  • Minutes of the CHMP Meeting 14-17 September 2020
    13 January 2021 EMA/CHMP/625456/2020 Corr.1 Human Medicines Division Committee for medicinal products for human use (CHMP) Minutes for the meeting on 14-17 September 2020 Chair: Harald Enzmann – Vice-Chair: Bruno Sepodes Disclaimers Some of the information contained in these minutes is considered commercially confidential or sensitive and therefore not disclosed. With regard to intended therapeutic indications or procedure scopes listed against products, it must be noted that these may not reflect the full wording proposed by applicants and may also vary during the course of the review. Additional details on some of these procedures will be published in the CHMP meeting highlights once the procedures are finalised and start of referrals will also be available. Of note, these minutes are a working document primarily designed for CHMP members and the work the Committee undertakes. Note on access to documents Some documents mentioned in the minutes cannot be released at present following a request for access to documents within the framework of Regulation (EC) No 1049/2001 as they are subject to on- going procedures for which a final decision has not yet been adopted. They will become public when adopted or considered public according to the principles stated in the Agency policy on access to documents (EMA/127362/2006). 1 Addition of the list of participants Official address Domenico Scarlattilaan 6 ● 1083 HS Amsterdam ● The Netherlands Address for visits and deliveries Refer to www.ema.europa.eu/how-to-find-us Send us a question Go to www.ema.europa.eu/contact Telephone +31 (0)88 781 6000 An agency of the European Union © European Medicines Agency, 2020.
    [Show full text]
  • Predictive QSAR Tools to Aid in Early Process Development of Monoclonal Antibodies
    Predictive QSAR tools to aid in early process development of monoclonal antibodies John Micael Andreas Karlberg Published work submitted to Newcastle University for the degree of Doctor of Philosophy in the School of Engineering November 2019 Abstract Monoclonal antibodies (mAbs) have become one of the fastest growing markets for diagnostic and therapeutic treatments over the last 30 years with a global sales revenue around $89 billion reported in 2017. A popular framework widely used in pharmaceutical industries for designing manufacturing processes for mAbs is Quality by Design (QbD) due to providing a structured and systematic approach in investigation and screening process parameters that might influence the product quality. However, due to the large number of product quality attributes (CQAs) and process parameters that exist in an mAb process platform, extensive investigation is needed to characterise their impact on the product quality which makes the process development costly and time consuming. There is thus an urgent need for methods and tools that can be used for early risk-based selection of critical product properties and process factors to reduce the number of potential factors that have to be investigated, thereby aiding in speeding up the process development and reduce costs. In this study, a framework for predictive model development based on Quantitative Structure- Activity Relationship (QSAR) modelling was developed to link structural features and properties of mAbs to Hydrophobic Interaction Chromatography (HIC) retention times and expressed mAb yield from HEK cells. Model development was based on a structured approach for incremental model refinement and evaluation that aided in increasing model performance until becoming acceptable in accordance to the OECD guidelines for QSAR models.
    [Show full text]
  • PARP Inhibitors Sensitize Ewing Sarcoma Cells to Temozolomide
    Published OnlineFirst October 5, 2015; DOI: 10.1158/1535-7163.MCT-15-0587 Cancer Biology and Signal Transduction Molecular Cancer Therapeutics PARP Inhibitors Sensitize Ewing Sarcoma Cells to Temozolomide-Induced Apoptosis via the Mitochondrial Pathway Florian Engert1, Cornelius Schneider1, Lilly Magdalena Weib1,2,3, Marie Probst1,and Simone Fulda1,2,3 Abstract Ewing sarcoma has recently been reported to be sensitive to that subsequent to DNA damage-imposed checkpoint activa- poly(ADP)-ribose polymerase (PARP) inhibitors. Searching for tion and G2 cell-cycle arrest, olaparib/TMZ cotreatment causes synergistic drug combinations, we tested several PARP inhibi- downregulation of the antiapoptotic protein MCL-1, followed by tors (talazoparib, niraparib, olaparib, veliparib) together with activation of the proapoptotic proteins BAX and BAK, mitochon- chemotherapeutics. Here, we report that PARP inhibitors syner- drial outer membrane permeabilization (MOMP), activation of gize with temozolomide (TMZ) or SN-38 to induce apoptosis caspases, and caspase-dependent cell death. Overexpression of a and also somewhat enhance the cytotoxicity of doxorubicin, nondegradable MCL-1 mutant or BCL-2, knockdown of NOXA or etoposide, or ifosfamide, whereas actinomycin D and vincris- BAX and BAK, or the caspase inhibitor N-benzyloxycarbonyl-Val- tine show little synergism. Furthermore, triple therapy of ola- Ala-Asp-fluoromethylketone (zVAD.fmk) all significantly reduce parib, TMZ, and SN-38 is significantly more effective compared olaparib/TMZ-mediated apoptosis. These findings emphasize with double or monotherapy. Mechanistic studies revealed that the role of PARP inhibitors for chemosensitization of Ewing the mitochondrial pathway of apoptosis plays a critical role in sarcoma with important implications for further (pre)clinical mediating the synergy of PARP inhibition and TMZ.
    [Show full text]
  • DRUGS REQUIRING PRIOR AUTHORIZATION in the MEDICAL BENEFIT Page 1
    Effective Date: 08/01/2021 DRUGS REQUIRING PRIOR AUTHORIZATION IN THE MEDICAL BENEFIT Page 1 Therapeutic Category Drug Class Trade Name Generic Name HCPCS Procedure Code HCPCS Procedure Code Description Anti-infectives Antiretrovirals, HIV CABENUVA cabotegravir-rilpivirine C9077 Injection, cabotegravir and rilpivirine, 2mg/3mg Antithrombotic Agents von Willebrand Factor-Directed Antibody CABLIVI caplacizumab-yhdp C9047 Injection, caplacizumab-yhdp, 1 mg Cardiology Antilipemic EVKEEZA evinacumab-dgnb C9079 Injection, evinacumab-dgnb, 5 mg Cardiology Hemostatic Agent BERINERT c1 esterase J0597 Injection, C1 esterase inhibitor (human), Berinert, 10 units Cardiology Hemostatic Agent CINRYZE c1 esterase J0598 Injection, C1 esterase inhibitor (human), Cinryze, 10 units Cardiology Hemostatic Agent FIRAZYR icatibant J1744 Injection, icatibant, 1 mg Cardiology Hemostatic Agent HAEGARDA c1 esterase J0599 Injection, C1 esterase inhibitor (human), (Haegarda), 10 units Cardiology Hemostatic Agent ICATIBANT (generic) icatibant J1744 Injection, icatibant, 1 mg Cardiology Hemostatic Agent KALBITOR ecallantide J1290 Injection, ecallantide, 1 mg Cardiology Hemostatic Agent RUCONEST c1 esterase J0596 Injection, C1 esterase inhibitor (recombinant), Ruconest, 10 units Injection, lanadelumab-flyo, 1 mg (code may be used for Medicare when drug administered under Cardiology Hemostatic Agent TAKHZYRO lanadelumab-flyo J0593 direct supervision of a physician, not for use when drug is self-administered) Cardiology Pulmonary Arterial Hypertension EPOPROSTENOL (generic)
    [Show full text]
  • AHFS Pharmacologic-Therapeutic Classification System
    AHFS Pharmacologic-Therapeutic Classification System Abacavir 48:24 - Mucolytic Agents - 382638 8:18.08.20 - HIV Nucleoside and Nucleotide Reverse Acitretin 84:92 - Skin and Mucous Membrane Agents, Abaloparatide 68:24.08 - Parathyroid Agents - 317036 Aclidinium Abatacept 12:08.08 - Antimuscarinics/Antispasmodics - 313022 92:36 - Disease-modifying Antirheumatic Drugs - Acrivastine 92:20 - Immunomodulatory Agents - 306003 4:08 - Second Generation Antihistamines - 394040 Abciximab 48:04.08 - Second Generation Antihistamines - 394040 20:12.18 - Platelet-aggregation Inhibitors - 395014 Acyclovir Abemaciclib 8:18.32 - Nucleosides and Nucleotides - 381045 10:00 - Antineoplastic Agents - 317058 84:04.06 - Antivirals - 381036 Abiraterone Adalimumab; -adaz 10:00 - Antineoplastic Agents - 311027 92:36 - Disease-modifying Antirheumatic Drugs - AbobotulinumtoxinA 56:92 - GI Drugs, Miscellaneous - 302046 92:20 - Immunomodulatory Agents - 302046 92:92 - Other Miscellaneous Therapeutic Agents - 12:20.92 - Skeletal Muscle Relaxants, Miscellaneous - Adapalene 84:92 - Skin and Mucous Membrane Agents, Acalabrutinib 10:00 - Antineoplastic Agents - 317059 Adefovir Acamprosate 8:18.32 - Nucleosides and Nucleotides - 302036 28:92 - Central Nervous System Agents, Adenosine 24:04.04.24 - Class IV Antiarrhythmics - 304010 Acarbose Adenovirus Vaccine Live Oral 68:20.02 - alpha-Glucosidase Inhibitors - 396015 80:12 - Vaccines - 315016 Acebutolol Ado-Trastuzumab 24:24 - beta-Adrenergic Blocking Agents - 387003 10:00 - Antineoplastic Agents - 313041 12:16.08.08 - Selective
    [Show full text]
  • Cancer Drug Pharmacology Table
    CANCER DRUG PHARMACOLOGY TABLE Cytotoxic Chemotherapy Drugs are classified according to the BC Cancer Drug Manual Monographs, unless otherwise specified (see asterisks). Subclassifications are in brackets where applicable. Alkylating Agents have reactive groups (usually alkyl) that attach to Antimetabolites are structural analogues of naturally occurring molecules DNA or RNA, leading to interruption in synthesis of DNA, RNA, or required for DNA and RNA synthesis. When substituted for the natural body proteins. substances, they disrupt DNA and RNA synthesis. bendamustine (nitrogen mustard) azacitidine (pyrimidine analogue) busulfan (alkyl sulfonate) capecitabine (pyrimidine analogue) carboplatin (platinum) cladribine (adenosine analogue) carmustine (nitrosurea) cytarabine (pyrimidine analogue) chlorambucil (nitrogen mustard) fludarabine (purine analogue) cisplatin (platinum) fluorouracil (pyrimidine analogue) cyclophosphamide (nitrogen mustard) gemcitabine (pyrimidine analogue) dacarbazine (triazine) mercaptopurine (purine analogue) estramustine (nitrogen mustard with 17-beta-estradiol) methotrexate (folate analogue) hydroxyurea pralatrexate (folate analogue) ifosfamide (nitrogen mustard) pemetrexed (folate analogue) lomustine (nitrosurea) pentostatin (purine analogue) mechlorethamine (nitrogen mustard) raltitrexed (folate analogue) melphalan (nitrogen mustard) thioguanine (purine analogue) oxaliplatin (platinum) trifluridine-tipiracil (pyrimidine analogue/thymidine phosphorylase procarbazine (triazine) inhibitor)
    [Show full text]
  • Classification Decisions Taken by the Harmonized System Committee from the 47Th to 60Th Sessions (2011
    CLASSIFICATION DECISIONS TAKEN BY THE HARMONIZED SYSTEM COMMITTEE FROM THE 47TH TO 60TH SESSIONS (2011 - 2018) WORLD CUSTOMS ORGANIZATION Rue du Marché 30 B-1210 Brussels Belgium November 2011 Copyright © 2011 World Customs Organization. All rights reserved. Requests and inquiries concerning translation, reproduction and adaptation rights should be addressed to [email protected]. D/2011/0448/25 The following list contains the classification decisions (other than those subject to a reservation) taken by the Harmonized System Committee ( 47th Session – March 2011) on specific products, together with their related Harmonized System code numbers and, in certain cases, the classification rationale. Advice Parties seeking to import or export merchandise covered by a decision are advised to verify the implementation of the decision by the importing or exporting country, as the case may be. HS codes Classification No Product description Classification considered rationale 1. Preparation, in the form of a powder, consisting of 92 % sugar, 6 % 2106.90 GRIs 1 and 6 black currant powder, anticaking agent, citric acid and black currant flavouring, put up for retail sale in 32-gram sachets, intended to be consumed as a beverage after mixing with hot water. 2. Vanutide cridificar (INN List 100). 3002.20 3. Certain INN products. Chapters 28, 29 (See “INN List 101” at the end of this publication.) and 30 4. Certain INN products. Chapters 13, 29 (See “INN List 102” at the end of this publication.) and 30 5. Certain INN products. Chapters 28, 29, (See “INN List 103” at the end of this publication.) 30, 35 and 39 6. Re-classification of INN products.
    [Show full text]
  • Comparison of Pharmacological Activity of Macitentan and Bosentan in Preclinical Models of Systemic and Pulmonary Hypertension
    LFS-13929; No of Pages 7 Life Sciences xxx (2014) xxx–xxx Contents lists available at ScienceDirect Life Sciences journal homepage: www.elsevier.com/locate/lifescie Comparison of pharmacological activity of macitentan and bosentan in preclinical models of systemic and pulmonary hypertension Marc Iglarz ⁎, Alexandre Bossu, Daniel Wanner, Céline Bortolamiol, Markus Rey, Patrick Hess, Martine Clozel Drug Discovery Department, Actelion Pharmaceuticals Ltd, Gewerbestrasse 16, 4123 Allschwil, Switzerland article info abstract Article history: Aims: The endothelin (ET) system is a tissular system, as the production of ET isoforms is mostly autocrine or Received 29 October 2013 paracrine. Macitentan is a novel dual ETA/ETB receptor antagonist with enhanced tissue distribution and Accepted 12 February 2014 sustained receptor binding properties designed to achieve a more efficacious ET receptor blockade. To determine Available online xxxx if these features translate into improved efficacy in vivo, a study was designed in which rats with either systemic or pulmonary hypertension and equipped with telemetry were given macitentan on top of maximally effective Keywords: doses of another dual ET /ET receptor antagonist, bosentan, which does not display sustained receptor occupan- Endothelin A B Pharmacology cy and shows less tissue distribution. – Blood pressure Main methods: After establishing dose response curves of both compounds in conscious, hypertensive Dahl salt- Pulmonary hypertension sensitive and pulmonary hypertensive bleomycin-treated rats, macitentan was administered on top of the max- Rat imal effective dose of bosentan. Key findings: In hypertensive rats, macitentan 30 mg/kg further decreased mean arterial blood pressure (MAP) by 19 mm Hg when given on top of bosentan 100 mg/kg (n =9,p b 0.01 vs.
    [Show full text]
  • Evkeeza™ (Evinacumab-Dgnb) NOTICE
    DRUG POLICY Evkeeza™ (evinacumab-dgnb) NOTICE This policy contains information which is clinical in nature. The policy is not medical advice. The information in this policy is used by Wellmark to make determinations whether medical treatment is covered under the terms of a Wellmark member's health benefit plan. Physicians and other health care providers are responsible for medical advice and treatment. If you have specific health care needs, you should consult an appropriate health care professional. If you would like to request an accessible version of this document, please contact customer service at 800-524-9242. BENEFIT APPLICATION Benefit determinations are based on the applicable contract language in effect at the time the services were rendered. Exclusions, limitations or exceptions may apply. Benefits may vary based on contract, and individual member benefits must be verified. Wellmark determines medical necessity only if the benefit exists and no contract exclusions are applicable. This policy may not apply to FEP. Benefits are determined by the Federal Employee Program. DESCRIPTION The intent of the Evkeeza™ policy is to provide coverage consistent with product labeling, FDA guidance, standards of medical practice, evidence-based drug information, and/or published guidelines. The indications below including FDA-approved indications and compendial uses are considered covered benefits provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy. FDA-Approved Indications Evkeeza (evinacumab-dgnb) is an ANGPTL3 (angiopoietin-like 3) inhibitor indicated as an adjunct to other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients, aged 12 years and older, with homozygous familial hypercholesterolemia (HoFH).
    [Show full text]
  • Opsumit) Reference Number: CP.PHAR.194 Effective Date: 03.16 Last Review Date: 02.21 Line of Business: Medicaid Revision Log
    Clinical Policy: Macitentan (Opsumit) Reference Number: CP.PHAR.194 Effective Date: 03.16 Last Review Date: 02.21 Line of Business: Medicaid Revision Log See Important Reminder at the end of this policy for important regulatory and legal information. Description Macitentan (Opsumit®) is an endothelin receptor antagonist. FDA Approved Indication(s) Opsumit is indicated for treatment of pulmonary arterial hypertension (PAH) (World Health Organization (WHO) Group I) to reduce the risks of disease progression and hospitalization for PAH. Effectiveness was established in a long-term study in PAH patients with predominantly WHO Functional Class II-III symptoms treated for an average of 2 years. Patients had idiopathic and heritable PAH (57%), PAH caused by connective tissue disorders (31%), and PAH caused by congenital heart disease with repaired shunts (8%). 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 Opsumit is medically necessary when the following criteria are met: I. Initial Approval Criteria A. Pulmonary Arterial Hypertension (must meet all): 1. Diagnosis of PAH; 2. Prescribed by or in consultation with a cardiologist or pulmonologist; 3. Failure of a calcium channel blocker (see Appendix B), unless member meets one of the following (a or b): a. Inadequate response or contraindication to acute vasodilator testing; b. Contraindication or clinically significant adverse effects to calcium channel blockers are experienced; 4. Failure of generic ambrisentan or bosentan, unless clinically significant adverse effects are experienced or both are contraindicated; 5.
    [Show full text]
  • New Drug Update: the Next Step in Personalized Medicine
    New Drug Update: The Next Step in Personalized Medicine Jordan Hill, PharmD, BCOP Clinical Pharmacy Specialist WVU Medicine Mary Babb Randolph Cancer Center Objectives • Review indications for new FDA approved anti- neoplastic medications in 2017 • Outline place in therapy of new medications • Become familiar with mechanisms of action of new medications • Describe adverse effects associated with new medications • Summarize dosing schemes and appropriate dose reductions for new medications First-in-Class Approvals • FLT3 inhibitor – midostaurin • IDH2 inhibitor – enasidenib • Anti-CD22 antibody drug conjugate – inotuzumab ozogamicin • CAR T-cell therapy - tisagenlecleucel “Me-too” Approvals • CDK 4/6 inhibitor – ribociclib and abemaciclib • PD-L1 inhibitors • Avelumab • Durvalumab • PARP inhibitor – niraparib • ALK inhibitor – brigatinib • Pan-HER inhibitor – neratinib • Liposome-encapsulated combination of daunorubicin and cytarabine • PI3K inhibitor – copanlisib Drugs by Malignancy AML Breast Bladder ALL FL Lung Ovarian 0 1 2 3 Oral versus IV 46% 54% Oral IV FIRST-IN CLASS FLT3 Inhibitor - midostaurin Journal of Hematology & Oncology 2017;10:93 Midostaurin (Rydapt®) • 717 newly diagnosed FLT3+ AML Patients • Induction and consolidation chemotherapy and placebo maintenance versus chemotherapy + midostaurin and Treatment maintenance midostaurin • OS: 74.7 mo versus 25.6 mo (P=0.009) Efficacy • EFS: 8.2 mo versus 3.0 mo (P=0.002) • Higher grade > 3 anemia, rash, and nausea Safety N Engl J Med 2017;377:454-64 Midostaurin (Rydapt®) • Approved indications: FLT3+ AML, mast cell leukemia, systemic mastocytosis • AML dose: 50 mg twice daily with food on days 8-21 • Of each induction cycle (+ daunorubicin and cytarabine) • Of each consolidation cycle (+ high dose cytarabine) • ADEs: nausea, myelosuppression, mucositis, increases in LFTs, amylase/lipase, and electrolyte abnormalities • Pharmacokinetics: hepatic metabolism, substrate of CYP 3A4; < 5% excretion in urine RYDAPT (midostaurin) [package insert].
    [Show full text]