REMEDIUMREMEDIUM1561-59362658-3534Joint-Stock Company Chicot90810.21518/1561-5936-2021-3-69-75Original ArticleStudy of the availability of gene therapeutic drugs in the Russian FederationKoshechkinKonstantin A.-RomanovPhilip A.-MokhovAlexander A.-KhokhlovAlexander L.-Sechenov First Moscow State Medical University (Sechenov University)Ministry of Health of the Russian FederationKutafin Moscow State Law UniversityYaroslavl State Medical University151220213697518032022Copyright © 2021,2021The authors studied the availability of gene therapy drugs in the Russian Federation on the basis of information on the permission of the medical use of drugs of this group in the world. Literature data and information about medicines approved by the FDA, EMA and the Ministry of Health of Russia were used. In general, the FDA registered only 13 drugs (46%) of the total approved for medical use in the world, 2 of them have already been withdrawn from the market, and 2 additional clinical trials are underway. In Europe, the EMA has approved 16 drugs for medical use (57%), with 4 of them already withdrawn. Most of the drugs were first approved by the FDA, and then, on average, a year later, were approved in the European market. A total of 4 drugs were approved in the European market and were not approved by the FDA at the time the data was requested. And only 1 drug, approved in the USA, is not registered in Europe. In the Russian Federation, two medicines are allowed, Neovasculgen (2011) and Spinraza (2019). This is only 7% of the total number of gene therapy drugs on the world market. Most of the drugs are intended for the treatment of orphan diseases and are cost expensive. This can explain the unevenness of their distribution across regions.gene therapy drugscirculation of medicinesgene medicationsgene therapyгенно-терапевтические препаратыобращение лекарственных средствлекарственные препаратыгенотерапия1.Misra S. Human gene therapy: a brief overview of the genetic revolution. J Assoc Physicians India. India; 2013;61(2):127–133.2.Axelsen T.M., Woldbye D.P.D. Gene therapy for Parkinson’s disease, an update. Journal of Parkinson’s Disease. 2018;8(2):195–215.3.Choong C.J., Baba K., Mochizuki H. Gene therapy for neurological disorders. Expert Opin Biol Ther. Taylor and Francis Ltd; 2016;16(2):143–159.4.Chamberlain J.R., Chamberlain J.S. Progress toward Gene Therapy for Duchenne Muscular Dystrophy. Molecular Therapy. American Society of Gene and Cell Therapy. 2017;25(5):1125–1131.5.Wirth T., Parker N., Ylä-Herttuala S. History of gene therapy. Gene. 2013;525(2):162–169.6.Ginn S.L. et al. Gene therapy clinical trials worldwide to 2017: An update. Journal of Gene Medicine. 2018;20(5).7.Al-Zaidy S.A., Mendell J.R. From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1. Pediatric Neurology. 2019;100:3–11.8.Peyvandi F., Garagiola I. Clinical advances in gene therapy updates on clinical trials of gene therapy in haemophilia. Haemophilia. 2019;25(5):738–746.9.Anguela X.M., High K.A. Entering the modern era of gene therapy. Annual Review of Medicine. 2019;70:273–288.10.Collins M., Thrasher A. Gene therapy: Progress and predictions. Proceedings of the Royal Society B: Biological Sciences. 2015;282(1821).11.Мохов А.А., Меркулов В.А., Мельникова Е.В., Попов П.И., Дудченко В.В., Кошечкин К.А. и др. Номенклатура генотерапевтических лекарственных препаратов: международный опыт. Ремедиум. 2019;(5):22–26. https://doi.org/10.21518/1561-5936-2019-5-22-26.12.В России испытают на пациентах препарат с геном-убийцей рака. Режим доступа: https://old.sk.ru/news/b/press/archive/2021/04/12/v-rossii-ispytayut-na-pacientah-preparat-s-genomubiycey-raka.aspx (accessed: 24.04.2021).13.«Биокад» готовит доклинические испытания генотерапевтического препарата от СМА – журнал Vademecum. Режим доступа: https://vademec.ru/news/2021/03/16/biokad-zavershil-razrabotku-otechestvennogopreparata-ot-sma-/ (accessed: 24.04.2021).14.Разработан препарат для персонифицированной генной терапии на основе клеток крови пациента. Режим доступа: https://www.rscf.ru/news/release/razrabotan-preparat-dlya-personifitsirovannoy-gennoy-terapii-naosnove-kletok-krovi-patsienta-/ (accessed: 24.04.2021).15.Мельникова Е.В. et al. Мировой опыт регистрации и применения препаратов для генной терапии в клинической практике. Антибиотики и химиотерапия. 2019;64(1–2):58–68.16.Highleyman L. Fomivirsen. BETA: bulletin of experimental treatments for AIDS: a publication of the San Francisco AIDS Foundation. United States; 1998:29–31.17.Zhang W.W. et al. The First Approved Gene Therapy Product for Cancer Ad-p53 (Gendicine): 12 Years in the Clinic. Hum Gene Ther. 2018;29(2):160–179.18.Chervyakov Y.V. et al. Five-year results of treating patients with chronic lower limb ischaemia by means of gene engineering. Angiol Sosud Khir. 2016;22(4):38–44.19.Watanabe N. et al. Re-examination of regulatory opinions in Europe: possible contribution for the approval of the first gene therapy product Glybera. Mol Ther Methods Clin Dev. 2015;2:14066.20.The million-dollar drug CBC News. Available at: https://newsinteractives.cbc.ca/longform/glybera (accessed: 04.11.2020).21.Goodbye Glybera! The World’s First Gene Therapy will be Withdrawn. Available at: https://www.labiotech.eu/medical/uniqure-glybera-marketing-withdrawn/ (accessed: 04.11.2020).22.Pacione M. et al. Perspectives on Spinraza (Nusinersen) Treatment Study: Views of Individuals and Parents of Children Diagnosed with Spinal Muscular Atrophy. J Neuromuscul Dis. 2019;6(1):119–131.23.Mercuri E. et al. Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy. N Engl J Med. 2018;378(7):625–635.24.Brudno J.N., Kochenderfer J.N. Recent advances in CAR T-cell toxicity: Mechanisms, manifestations and management. Blood Reviews. 2019;34:45–55.25.Neelapu S.S. Managing the toxicities of CAR T-cell therapy. Hematol Oncol. 2019;37(S1):48–52.26.Vormittag P. et al. A guide to manufacturing CAR T cell therapies. Current Opinion in Biotechnology. 2018;53:164–181.27.Hoy S.M. Onasemnogene Abeparvovec: First Global Approval. Drugs. 2019;79(11):1255–1262.28.Synthetic Biology Investment Reached a New Record of Nearly $8 Billion in 2020 — What Does This Mean For 2021? – SynBioBeta. Available at: https://synbiobeta.com/synthetic-biology-investment-set-a-nearly-8-billion-record-in-2020-what-does-this-mean-for-2021 (accessed: 10.04.2021).29.Как получить незарегистрированные лекарства — Про Паллиатив. Режим доступа: https://pro-palliativ.ru/blog/kak-poluchit-nezaregistrirovannye-lekarstva (accessed: 04.11.2020).30.В России создадут Федеральный центр планирования и организации лекарственного обеспечения граждан. Режим доступа: https://minzdrav.gov.ru/news/2020/11/03/15337-v-rossii-sozdadut-federalnyy-tsentrplanirovaniya-i-organizatsii-lekarstvennogo-obespecheniya-grazhdan (accessed: 10.04.2021).31.«Круг добра» закупил первые препараты для пациентов со СМА. Российская Фармацевтика. https://pharmapractice.ru/174070 (accessed: 10.04.2021).32.Biocad завершила раннюю разработку первого российского лекарства от СМА. Режим доступа: https://www.forbes.ru/newsroom/tehnologii/423479-biocadzavershila-rannyuyu-razrabotku-pervogo-rossiyskogo-lekarstva-ot (accessed: 10.04.2021).33.Отечественный препарат от СМА готов к доклиническим испытаниям – журнал Vademecum. Режим доступа: https://vademec.ru/news/2020/02/29/rossiyskaya-kompaniya-razrabatyvaet-preparat-ot-spinalnoy-myshechnoy-atrofii/ (accessed: 24.04.2021).34.«Генериум» начал КИ орфанного препарата с объемом рынка свыше 2 млрд – журнал Vademecum. Режим доступа: https://vademec.ru/news/2019/12/04/generium-nachal-ki-orfannogo-preparata-s-obemom-rynkasvyshe-2-mlrd/ (accessed: 24.04.2021).35.Рынок получил шанс на медикаментозное ускорение: Общество: Газета РБК. Режим доступа: https://www.rbc.ru/newspaper/2021/03/16/6049c9d39a79478f448481c9 (accessed: 10.04.2021).36.Олефир Ю.В., Меркулов В.А., Кошечкин К.А. Переход к электронной регистрации лекарственных средств для единого фармацевтического рынка евразийского экономического союзаю. Ремедиум. 2017;(6):42–46. https://doi.org/10.21518/1561-5936-2017-6-42-46.37.Кошечкин К.А., Рычихина Е.М., Олефир Ю.В. Цифровые системы для формирования электронного досье. Ремедиум. 2018;(10):37–41. https://doi.org/10.21518/1561-5936-2018-10-37-41.