Nirogacestat in treatment of patients with desmoid tumors. Literature review

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Abstract

This literature review analyzes the role of the gamma-secretase inhibitor nirogacestat in treating patients with progressive desmoid tumors. The article systematizes data on the molecular mechanisms of the drug's action, stages of its clinical development, and the final results of the phase III DeFi trial, which formed the basis for nirogacestat's regulatory approval. It is shown that the drug significantly improves progression-free survival, increases objective response rate, and leads to clinically meaningful improvement in quality of life. The review discusses nirogacestat's safety profile in detail, highlighting a specific yet reversible adverse event – ovarian dysfunction. The analyzed data provide evidence for the efficacy and safety of nirogacestat, supporting its consideration as a new therapeutic option for patients with progressive desmoid fibromatosis.

About the authors

K. A. Turupaev

N.N. Blokhin National Medical Research Center of Oncology, Ministry of Health of Russia

Author for correspondence.
Email: Kir-turupaev@yandex.ru
ORCID iD: 0000-0001-8887-5108
Russian Federation, 24 Kashirskoe Shosse, Moscow, 115522

O. P. Grebennikova

Russian University of Medicine, Ministry of Health of Russia

Email: Kir-turupaev@yandex.ru
ORCID iD: 0009-0007-1861-3306
Russian Federation, 4 Dolgorukovskaya St., Moscow, 127006

M. D. Budurova

Russian University of Medicine, Ministry of Health of Russia; P.A. Hertzen Moscow Oncology Research Institute – branch of the National Medical Research Radiological Center, Ministry of Health of Russia

Email: Kir-turupaev@yandex.ru
ORCID iD: 0000-0003-1391-0516
Russian Federation, 4 Dolgorukovskaya St., Moscow, 127006; 3 2nd Botkinskiy Proezd, Moscow, 125284

D. D. Ladyko

N.N. Blokhin National Medical Research Center of Oncology, Ministry of Health of Russia

Email: Kir-turupaev@yandex.ru
ORCID iD: 0009-0009-5878-6951
Russian Federation, 24 Kashirskoe Shosse, Moscow, 115522

V. Y. Selchuk

Russian University of Medicine, Ministry of Health of Russia

Email: Kir-turupaev@yandex.ru
Russian Federation, 4 Dolgorukovskaya St., Moscow, 127006

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