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| DESCRIPTION | - PYRUVATE KINASE-R, "Healthy Subjects", 56th Annual Meeting of the Am. Chem. Soc. Hematol.; San Francisco, CA, USA, (20140609), ISSN 0006-4971 | - |
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| EXAMINATION | - BLOOD, 56TH ANNUAL MEETING OF THE AMERICAN-SOCIETY-OF-HEMATOLOGY; SAN FRANCISCO, CA, USA; DECEMBER 06 -09, 2014, (201412), vol. 124, no. 21, ISSN 0006-4971(print) | - |
| EXAMINATION | - KUNG CHARLES ET AL, "AG-348 Activation of Pyruvate Kinase in Vivo Enhances Red Cell Glycolysis in Mice", BIOSIS, BIOSCIENCES INFORMATION SERVICE, PHILADELPHIA, PA, US, (201412), Database accession no. PREV201500280942, XP002787328 | - |
| EXAMINATION | - YANG HUA ET AL, "Phase I Single (SAD) and Multiple Ascending Dose (MAD) Studies of the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of AG-348, a First-in-Class Allosteric Activator of Pyruvate Kinase-R, in Healthy Subjects", BIOSIS, BIOSCIENCES INFORMATION SERVICE, PHILADELPHIA, PA, US, (201412), Database accession no. PREV201500280858, XP002787329 | - |
| INTERNATIONAL-SEARCH-REPORT | - ANASTASIOU et al., "Pyruvate kinase M2 activators promote tetramer formation and suppress tumorigenesis", Nat Chem Biol., (20120000), vol. 8, no. 10, pages 839 - 847, XP055333200 [Y] 23, 29-30 * . pg 9, para 4; pg 14, para 3 * | - |
| OPPOSITION | - "Agios Pharmaceuticals Announces Orphan Drug Designation of AG-348 for Treatment of Pyruvate Kinase Deficiency", Agios, (20150324), Agios, URL: https://investor.agios.com/node/7561/pdf, XP093300684 | - |
| OPPOSITION | - Allen, Loyd V. ; Popovich, Nicholas G. ; Ansel, Howard C. , "Chapter 2: New Drug Development and Approval Process", Allen, Loyd V. ; Popovich, Nicholas G. ; Ansel, Howard C. , Allen, Loyd V. ; Popovich, Nicholas G. ; Ansel, Howard C., [Pharmaceutical dosage forms and drug delivery systems] Ansel's pharmaceutical dosage forms and drug delivery systems, US , Lippincott Williams & Wilkins , (20050101), pages 25 - 66, ISBN 0-7817-4612-4, XP009556378 | - |
| OPPOSITION | - Anna Conrey, Ingrid Frey, Nancy Asomaning, Ruth Pierre-Charles, Dina Parekh, Julia Z. Xu, Kang Le , Britney Kruah, Quan Li, Emily Dunkelberger, Troy Cellmer, AmberYates, Megan Wind-Rotolo, Neal Jeffries, William A. Eaton, Swee Lay Thein, "Long-term Safety and Efficacy of Mitapivat, an Oral Pyruvate Kinase Activator, in Adults with Sickle Cell Disease: Extension of a Phase 1 Dose Escalation Study", 65th American Society of Hematology Annual Meeting, (20231209), pages 1 - 13, XP093234790 | - |
| OPPOSITION | - Anonymous, "Agios Announces FDA Approval of PYRUKYND® (mitapivat) as First Disease-Modifying Therapy for Hemolytic Anemia in Adults with Pyruvate Kinase Deficiency", Agios, (20220217), pages 1 - 4, Agios , URL: https://investor.agios.com/news-releases/news-release-details/agios-announces-fda-approval-pyrukyndr-mitapivat-first-disease, (20241216), XP093234763 | - |
| OPPOSITION | - Anonymous, "Agios Announces Phase 3 ENERGIZE-T Study of Mitapivat Met Primary Endpoint and All Key Secondary Endpoints in Adults with Transfusion-Dependent Alpha-or Beta-Thalassemia", Agios, (20240603), pages 1 - 3, Agios , URL: https://investor.agios.com/news-releases/news-release-details/agios-announces-phase-3-energize-t-study-mitapivat-met-primary, (20241216), XP093234787 | - |
| OPPOSITION | - Anonymous, "Agios Presents Positive Results from Phase 3 ENERGIZE Study of Mitapivat in Non-Transfusion-Dependent Thalassemia in Plenary Session at the European Hematology Association 2024 Hybrid Congress", Agios, (20240615), pages 1 - 3, Agios , URL: https://investor.agios.com/news-releases/news-release-details/agios-presents-positive-results-phase-3-energize-study-mitapivat, (20241216), XP093234782 | - |
| OPPOSITION | - Anonymous, "A Phase 2, Open Label, Randomized, Dose Ranging, Safety, Efficacy, Pharmacokinetic and Pharmacodynamic Study of AG348 in Adult Patients with Pyruvate Kinase Deficiency", EU Clinical Trials Register; 2015-000484-13, (20150518), EU Clinical Trials Register; 2015-000484-13, URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-000484-13/GB, XP093193074 | - |
| OPPOSITION | - Anonymous, "EMA recommends suspension of sickle cell disease medicine Oxbryta", EMA European medicines agency, (20240926), pages 1 - 5, EMA European medicines agency , URL: https://www.ema.europa.eu/en/news/ema-recommends-suspension-sickle-cell-disease-medicine-oxbryta, (20241216), XP093234817 | - |
| OPPOSITION | - Anonymous, "FDA is alerting patients and health care professionals about the voluntary withdrawal of Oxbryta from the market due to safety concerns", FDA - U.S food & drug administration, (20240926), pages 1 - 2, FDA - U.S food & drug administration, URL: https://www.fda.gov/drugs/drug-safety-and-availability/fda-alerting-patients-and-health-care-professionals-about-voluntary-withdrawal-oxbryta-market-due, (20241216), XP093234818 | - |
| OPPOSITION | - Anonymous, "ICH Topic E 4 - Dose Response Information to Support Drug Registration", European Medicines Agency; CPMP/ICH/378/95, (19941101), European Medicines Agency; CPMP/ICH/378/95, URL: https://www.ema.europa.eu/en/documents/scientific-guideline/ich-e-4-dose-response-information-support-drug-registration-step-5_en.pdf, (20240411), XP093150470 | - |
| OPPOSITION | - Anonymous, "International Nonproprietary Names for Pharmaceutical Substances (INN)", WHO Drug Information, (20160101), vol. 30, no. 4, pages 608 - 710, XP093234825 | - |
| OPPOSITION | - Anonymous, "PYRUKYND (mitapivat) Approved in the EU for the Treatment of Pyruvate Kinase (PK) Deficiency in Adult Patients", Agios, (20221110), pages 1 - 3, Agios , URL: https://investor.agios.com/news-releases/news-release-details/pyrukyndr-mitapivat-approved-eu-treatment-pyruvate-kinase-pk, (20241216), XP093234765 | - |
| OPPOSITION | - Anonymous, "REBLOZYL® (LUSPATERCEPT-AAMT) AND ITS MECHANISM OF ACTION", Bristol Myers Squibb, (20230101), pages 1 - 3, Bristol Myers Squibb, URL: https://mms.businesswire.com/media/20230622657196/en/1876772/1/Reblozyl_MOA_Fact_Sheet_082823_JK.pdf, XP009558859 | - |
| OPPOSITION | - Anonymous, "Recommended Tips for Creating an Orphan Drug Designation Application", FDA (A Webinar by the Office of Orphan Products Development (OOPD)), (20180101), FDA (A Webinar by the Office of Orphan Products Development (OOPD)), URL: https://www.fda.gov/media/111762/download, XP093300686 | - |
| OPPOSITION | - Anonymous, "Topline Results: Phase 3 ENERGIZE Study of PYRUKYND (mitapivat) in Thalassemia", Agios, (20240103), pages 1 - 20, Agios , URL: https://investor.agios.com/static-files/739fe687-5277-41b8-9a7c-575ba14e2539, (20241216), XP093234781 | - |
| OPPOSITION | - Bowden Chris, "FDA grants orphan drug designation to AG-348 for pyruvate kinase deciency ", Healio, (20150325), Healio, URL: https://www.healio.com/news/hematology-oncology/20150325/fda-grants-orphan-drug-designation-to-ag348-for-pyruvate-kinase-deficiency, XP093300682 | - |
| OPPOSITION | - D44a - Revised Table of main and auxiliary requests | - |
| OPPOSITION | - D45a-l - Excerpts of references 1-12 | - |
| OPPOSITION | - D45 - Expert Opinion of Mr. Robin Blaney, MA, including the CV, a copy of D5 | - |
| OPPOSITION | - D48 - FDA's Orphan Drug Designations and Approvals database extract Mitapivat (no date found) | - |
| OPPOSITION | - D49 - FDA's Orphan Drug Designations and Approvals database extract Mitapivat for PKD | - |
| OPPOSITION | - D51 - PYRUKYND® information, URL: https://www.pyrukynd.com/taking-pyrukynd/ | - |
| OPPOSITION | - Dhaliwal Gurpreet, Patricia A. Cornett, Lawrence M. Tierney, "Hemolytic Anemia", American Family Physician, (20040601), vol. 69, no. 11, pages 2599 - 2606, XP093193076 | - |
| OPPOSITION | - Grace Rachael F, D Mark Layton, Frederic Galacteros, Christian Rose, Wilma Barcellini, D Holmes Morton, Eduard Van Beers, Hassan Yaish, Yaddanapudi Ravindranath, Kevin Hm Kuo, Sujit Sheth, Janet L Kwiatkowski, Bruce Silver, Charles Kung, Varsha Iyer, Hua Yang, Penelope A Kosinski, Lei Hua, Ann Barbier, Bertil Glader, "Effects of AG-348, a pyruvate kinase activator, in patients with pyruvate kinase deficiency: Updated results from the DRIVE PK study D3", 59th American Society of Hematology (ASH) Annual Meeting, (20171209), pages 1 - 1, XP093234770 | - |
| OPPOSITION | - Kung Charles, Hill Collin, Chen Yue, Jha Abhishek, Kosinski Penelope, Clasquin Michelle, Si Yaguang, Kim Hyeryun, Hixon Jeff, Dang Lenny, Agresta Sam, Silverman Lee, Yang Hua, "AG-348 Activation of Pyruvate Kinase in Vivo Enhances Red Cell Glycolysis in Mice", The 56th American Society of Hematology Annual Meeting and Exposition, US , (20141206), pages 1 - 1, XP093234703 | - |
| OPPOSITION | - Kuo Kevin H, Layton D Mark, Lal Ashutosh, Ai-Samkari Hanny, Bhatia Joy, Tong Bo, Lynch Megan, Uhlig Katrin, Vichinsky Elliott P, "Results from a phase 2, open-label, multicenter study of the oral pyruvate kinase activator mitapivat in adults with non-transfusion-dependent alpha-or beta-thalassemia", European Hematology Association (EHA), (20210709), pages 1 - 15, XP093234777 | - |
| OPPOSITION | - Ldowu Modupe, Otieno Lucas, Dumitriu Bogdan, Lobo Clarisse Lc, Thein Swee Lay, Andemariam Biree, Nnodu E, Mo, Adlette, Glaros Alexander K, Teresinha Sara, La Saad Ola /, Bartolucci Pablo, Colombatti, Mo ', Taher Ali T, Mdphdfrcp ', Abboud Miguel R, Oluyadi Abdulafeez, Iyer Varsha, Yin Ophelia, Pho, Morris Susan, Yates Amber M, Shao Hui, Pho ', Patil Spurthi, Urbstonaitis Rolandas, Zaidi Ahmar U, Smith Wally R, "114. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Clinical and Epidemiological", Blood - The 65th ASH Annual Meeting Abstracts, (20230101), vol. 142, pages 271 - 273, XP093234805 | - |
| OPPOSITION | - Maria Almira Correia, "Chapter 4: Drug Biotransformation", Basic & Clinical Pharmacology, US, McGraw-Hill Medical, (20120101), pages 53 - 68, ISBN 978-0-07-176402-5, XP009555926 | - |
| OPPOSITION | - Nicholas H. G. Holford, "Chapter 3: Pharmacokinetics & Pharmacodynamics: Rational Dosing & the Time Course of Drug Actio", Basic & Clinical Pharmacology, US, McGraw-Hill Medical, (20120101), pages 37 - 51, ISBN 978-0-07-176402-5, XP009555925 | - |
| OPPOSITION | - "Orphan Drug Regulations", (20130612), vol. 78, no. 113, pages 35117 - 35135, XP093314072 | - |
| OPPOSITION | - R Schmidt, "Dose-Finding Studies in Clinical Drug Development", European Journal of Clinical Pharmacology, (19880101), vol. 34, pages 15 - 19, XP055350888 | - |
| OPPOSITION | - Van Dijk Myrthe J, Rab Minke A E, Van Oirschot Brigitte A, Bos Jennifer, Derichs Cleo, Rijneveld Anita W, Cnossen Marjon H, Nur Erfan, Biemond Bart J, Barte1s Marije, Jans Judith J M, Van Solinge Wouter W, Schutgens Roger E G, Van Wijk Richard, Van Beers Eduard J, "One-year follow-up of a phase 2 study of mitapivat, an oral pyruvate kinase activator, for the treatment of sickle cell disease", UMC Utrecht, (20230101), pages 1 - 1, XP093234800 | - |
| OPPOSITION | - von Zastrow, Mark, "Chapter 2: Drug Receptors / Pharmacodynamics", Basic & Clinical Pharmacology, US, McGraw-Hill Medical, (20120101), pages 15 - 35, ISBN 978-0-07-176401-8, XP009555924 | - |
| OPPOSITION | - Zerez Charles R, Lachant Neil A, Tanaka Kouichi R, , "Decreased erythrocyte phosphoribosylpyrophosphate synthetase activity and impaired formation in thalassemia minor: A mechanism for decreased adenine nucleotide content", Journal of Laboratory and Clinical Medicine, (19890701), vol. 114, no. 1, pages 43 - 50, XP093234739 | - |
| OPPOSITION | - Hua Yang; Elizabeth Merica; Yue Chen; Marvin Cohen; Ronald Goldwater; Penelope A. Kosinski; Charles Kung; Zheng (Jason) Yuan; Lee Silverman; Meredith Goldwasser; Bruce A. Silver; Sam Agresta; Ann J. Barbier, "Phase 1 Single‐ and Multiple‐Ascending‐Dose Randomized Studies of the Safety, Pharmacokinetics, and Pharmacodynamics of AG‐348, a First‐in‐Class Allosteric Activator of Pyruvate Kinase R, in Healthy Volunteers", Clinical Pharmacology in Drug Development, Wiley-Blackwell Publishing Ltd., GB, GB , (20180809), vol. 8, no. 2, doi:10.1002/cpdd.604, ISSN 2160-763X, pages 246 - 259, XP072461903 | |
| OPPOSITION | - Indrani Chakraborty; Raghwendra Mishra; Ratan Gachhui; Manoj Kar, "Distortion of -globin Chain of Hemoglobin Alters the Pathway of Erythrocytic Glucose Metabolism Through Band 3 Protein", Archives of medical research, MX , (20120125), vol. 43, no. 2, doi:10.1016/j.arcmed.2012.02.003, ISSN 0188-4409, pages 112 - 116, XP028483883 | |
| OPPOSITION | - Daughton Christian G., Ruhoy Ilene Sue, "Lower-dose prescribing: Minimizing “side effects” of pharmaceuticals on society and the environment", Science of The Total Environment, Elsevier, AMSTERDAM, NL, AMSTERDAM, NL , (20121129), vol. 443, doi:10.1016/j.scitotenv.2012.10.092, ISSN 0048-9697, pages 324 - 337, XP055939297 | |
| OPPOSITION | - Shaeffer Joseph R, "Turnover of Excess Hemoglobin (I! Chains in &Thalassemic Cells Is ATP-dependent", The journal of biological chemistry, (19831101), vol. 258, no. 21, doi:10.1016/S0021-9258(17)44097-X, pages 13172 - 13177, XP093234710 | |
| OPPOSITION | - Shaeffer Joseph R, "ATP-dependent Proteolysis of Hemoglobin alpha Chains in beta-Thalassemic Hemolysates Is Ubiquitin-dependent", The journal of biological chemistry, (19880901), vol. 263, no. 27, doi:10.1016/S0021-9258(18)68292-4, pages 13663 - 13669, XP093234719 | |
| OPPOSITION | - Betz Timo, Lenz Martin, Joanny Jean-François, Sykes Cécile, "ATP-dependent mechanics of red blood cells", Proceedings of the National Academy of Sciences (PNAS), (20090908), vol. 106, no. 36, doi:10.1073/pnas.0904614106, ISSN 0027-8424, pages 15320 - 15325, XP093234692 | |
| OPPOSITION | - Park Yongkeun, Best Catherine A., Auth Thorsten, Gov Nir S., Safran Samuel A., Popescu Gabriel, Suresh Subra, Feld Michael S., "Metabolic remodeling of the human red blood cell membrane", Proceedings of the National Academy of Sciences (PNAS), (20100126), vol. 107, no. 4, doi:10.1073/pnas.0910785107, ISSN 0027-8424, pages 1289 - 1294, XP093234696 | |
| OPPOSITION | - Steinberg Martin H., "Sickle Cell Anemia, the First Molecular Disease: Overview of Molecular Etiology, Pathophysiology, and Therapeutic Approaches", THE SCIENTIFIC WORLD JOURNAL, The ScientificWorld Ltd, GB, NL, GB, NL , (20080101), vol. 8, doi:10.1100/tsw.2008.157, ISSN 1537-744X, pages 1295 - 1324, XP093193077 | |
| OPPOSITION | - Scott G. L., Rasbridge M. R., Grimes A. J., "In Vitro Studies of Red Cell Metabolism in Haemoglobin H Disease", British Journal of Haematology, Hoboken, USA, (19700101), vol. 18, no. 1, doi:10.1111/j.1365-2141.1970.tb01415.x, ISSN 0007-1048, pages 13 - 28, XP093234729 | |
| OPPOSITION | - Richard L. Sabina; Nancy J. Wandersee; Cheryl A. Hillery, "Ca2+‐CaM activation of AMP deaminase contributes to adenine nucleotide dysregulation and phosphatidylserine externalization in human sickle erythrocytes", British Journal of Haematology, Hoboken, USA, (20081119), vol. 144, no. 3, doi:10.1111/j.1365-2141.2008.07473.x, ISSN 0007-1048, pages 434 - 445, XP071161841 | |
| OPPOSITION | - Weed R. I., Lacelle P. L., Merrill E. W., "Metabolic dependence of red cell deformability", Journal of Clinical Investigation, (19690101), vol. 48, no. 5, doi:10.1172/JCI106038, ISSN 0021-9738, pages 795 - 809, XP093234682 | |
| OPPOSITION | - Charache Samuel, Grisolia Santiago, Fiedler Adam J., Hellegers Andre E., "Effect of 2,3-diphosphoglycerate on oxygen affinity of blood in sickle cell anemia", Journal of Clinical Investigation, (19700401), vol. 49, no. 4, doi:10.1172/JCI106294, ISSN 0021-9738, pages 806 - 812, XP093234743 | |
| OPPOSITION | - Rachmilewitz Eliezer A., Giardina Patricia J., "How I treat thalassemia", Blood, American Society of Hematology, US, US , (20110929), vol. 118, no. 13, doi:10.1182/blood-2010-08-300335, ISSN 0006-4971, pages 3479 - 3488, XP093193075 | |
| OPPOSITION | - Khandros Eugene; Thom Christopher S.; D'Souza Janine; Weiss Mitchell J., "Integrated protein quality-control pathways regulate free α-globin in murine β-thalassemia", Blood, US , (20120531), vol. 119, no. 22, doi:10.1182/blood-2011-12-397729, ISSN 0006-4971, pages 5265 - 5275, XP086694036 | |
| OPPOSITION | - Yang Hua; Merica Elizabeth; Chen Yue; Cohen Marvin; Goldwater Ronald; Hill Collin; Kim Hyeryun; Kosinski Penelope; Kung Charles; Silver Bruce; Utley Luke; Agresta Sam, "Phase I Single (SAD) and Multiple Ascending Dose (MAD) Studies of the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of AG-348, a First-in-Class Allosteric Activator of Pyruvate Kinase-R, in Healthy Subjects", Blood, American Society of Hematology, US, US , (20141114), vol. 124, no. 21, doi:10.1182/blood.V124.21.4007.4007, ISSN 0006-4971, page 4007, XP086742812 | |
| OPPOSITION | - Michael Jensen, Stephen B. Shohet , David G. Nathan, "The Role of Red Cell Energy Metabolism in the Generation of Irreveribly Sickled Cells In Vitro", Blood, (19731201), vol. 42, no. 6, doi:10.1182/blood.V42.6.835.835, pages 835 - 842, XP093234753 | |
| OPPOSITION | - Poillon Wn, Kim Bc, Labotka Rj, Hicks Cu, Kark Ja, "Antisickling effects of 2,3-diphosphoglycerate depletion", Blood, US , (19950601), vol. 85, no. 11, doi:10.1182/blood.V85.11.3289.bloodjournal85113289, ISSN 0006-4971, pages 3289 - 3296, XP093234747 | |
| SEARCH | [ ] - BLOOD, 56TH ANNUAL MEETING OF THE AMERICAN-SOCIETY-OF-HEMATOLOGY; SAN FRANCISCO, CA, USA; DECEMBER 06 -09, 2014, (201412), vol. 124, no. 21, ISSN 0006-4971(print) | - |
| SEARCH | - KUNG CHARLES ET AL, "AG-348 Activation of Pyruvate Kinase in Vivo Enhances Red Cell Glycolysis in Mice", BIOSIS, BIOSCIENCES INFORMATION SERVICE, PHILADELPHIA, PA, US, (201412), Database accession no. PREV201500280942, XP002787328 [X] 1-4 * abstract * [Y] 11-14 | - |
| SEARCH | - YANG HUA ET AL, "Phase I Single (SAD) and Multiple Ascending Dose (MAD) Studies of the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of AG-348, a First-in-Class Allosteric Activator of Pyruvate Kinase-R, in Healthy Subjects", BIOSIS, BIOSCIENCES INFORMATION SERVICE, PHILADELPHIA, PA, US, (201412), Database accession no. PREV201500280858, XP002787329 [Y] 1-14 * abstract * | - |
