Ваморолон – одобренный мировыми регуляторами глюкокортикостероид для терапии прогрессирующей мышечной дистрофии Дюшенна. Обзор литературы

Прогрессирующая мышечная дистрофия (ПМД) Дюшенна – X-сцепленное наследственное заболевание, проявляющееся в раннем детстве и прогрессирующее с возрастом, приводящее к потере возможности двигаться, дыхательной и сердечной недостаточности. Общепризнанным мировым «золотым стандартом» лечения ПМД Дюшенна являются глюкокортикостероиды. Однако до последнего времени только дефлазакорт (Эмфлаза®) имел одобрение Управления по санитарному надзору за качеством пищевых продуктов и медикаментов США (Food and Drug Administration, FDA) по показанию «ПМД Дюшенна». Все остальные препараты этой группы применялись off-label. В конце 2023 г. FDA, а затем и Европейское агентство лекарственных средств (European Medicines Agency, ЕМА) зарегистрировали еще один препарат из группы глюкокортикостероидов – ваморолон (Агамри®, от англ. Agamree®). А в 2024 г. ваморолон получил одобрение в Великобритании и Китае. Таким образом, ваморолон в настоящее время является признанным всеми ведущими мировыми регуляторами препаратом из группы глюкокортикостероидов для терапии ПМД Дюшенна.

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