vedomostiregmedРегуляторные исследования и экспертиза лекарственных средствRegulatory Research and Medicine Evaluation3034-30623034-3453Federal State Budgetary Institution ‘Scientific Centre for Expert Evaluation of Medicinal Products’ of the Ministry of Health of the Russian Federation (FSBI ‘SCEEMP’)vedomostiregmed-24Research ArticleОБЗОРНЫЕ И ПРОБЛЕМНЫЕ СТАТЬИGENERAL AND TOPICAL ARTICLESАлипоген типарвовек: долгая дорога к оценке отношения пользы и риска генотерапевтического препаратаAlipogene tiparvovec: a long journey of risk-benefit ratio assessment of gene therapy productsАляутдинР. Н.AlyautdinR. N.Alyautdin@expmed.ruРомановБ. К.RomanovB. K.noemail@neicon.ruПереверзевА. П.PereverzevA. P.noemail@neicon.ruЧикалоА. О.ChikaloA. O.noemail@neicon.ruБунятянН. Д.BunyatyanN. D.noemail@neicon.ruМеркуловВ. А.MerkulovV. A.noemail@neicon.ruМироновА. Н.MironovA. N.noemail@neicon.ruФедеральное государственное бюджетное учреждение «Научный центр экспертизы средств медицинского применения» Министерства здравоохранения Российской ФедерацииРоссияFederal State Budgetary Institution «Scientific Centre for Expert Evaluation of Medicinal Products» of the Ministry of Health of the Russian FederationRussian Federation201513022018013134Copyright © Аляутдин Р.Н., Романов Б.К., Переверзев А.П., Чикало А.О., Бунятян Н.Д., Меркулов В.А., Миронов А.Н., 20182018Аляутдин Р.Н., Романов Б.К., Переверзев А.П., Чикало А.О., Бунятян Н.Д., Меркулов В.А., Миронов А.Н.Alyautdin R.N., Romanov B.K., Pereverzev A.P., Chikalo A.O., Bunyatyan N.D., Merkulov V.A., Mironov A.N.Данная работа распространяется под лицензией Creative Commons Attribution 4.0.This work is licensed under a Creative Commons Attribution 4.0 License.https://www.vedomostincesmp.ru/jour/article/view/24

Согласно определению ЕМА, в число средств передовой терапии включены препараты, содержащие нуклеиновые кислоты с целью замещения или восстановления генетического дефекта. Алипоген типарвовек - лекарственный препарат, содержащий генетический материал, для лечения наследственного дефицита липопротеинлипазы, соответствует этим критериям. В обзоре рассмотрены патофизиологические принципы действия препарата в условиях генетического дефекта липопротеинлипазы. Представлены результаты доклинических исследований на мышах и кошках. Клинические исследования препарата показали его эффективность при наследственном дефиците липопротеинлипазы, однако действие препарата было непродолжительным. Вместе с тем, полученные данные позволили ЕМА разрешить применение алипогена типарвовка в клинической практике.

According to EMA, advanced therapy medicinal products include, inter alia, therapeutic systems containing nucleic acids used for replacement or repair of genetic defects. Alipogen tiparvovec, a medicine containing genetic material for treatment of hereditary deficiency of lipoprotein lipase, meets these criteria. The article summarizes pathophysiological principles of this medicine action in subjects with lipoprotein lipase deficiency. The article describes the results of preclinical research conducted in mice and cats. Clinical trials revealed its efficiency in patients with hereditary lipoprotein lipase deficiency, however the period during which the drug proved efficient was rather short. Nevertheless the data obtained from alipogen tiparvovec clinical trials were convincing enough for EMA to permit its application in clinical practice.

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The authors declare that there are no conflicts of interest present.