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The Desmoid Tumor Research Foundation

August 2025

Dermatologic adverse events associated with gamma secretase inhibitor nirogacestat: A retrospective multicenter cohort study

Author Information

Grant J Riew 1, Lauren M Guggina 2, Federico Repetto 3, Christine G Lian 3, Alexandra P Charrow 1, Nicole R LeBoeuf 2, Connie R Shi 4

1Harvard Medical School, Boston, Massachusetts; Department of Dermatology, Brigham and Women's Hospital, Boston, Massachusetts. 2Harvard Medical School, Boston, Massachusetts; Department of Dermatology, Brigham and Women's Hospital, Boston, Massachusetts; Department of Cutaneous Oncology, Dana Farber Cancer Institute, Boston, Massachusetts. 3Harvard Medical School, Boston, Massachusetts; Department of Pathology, Brigham and Women's Hospital, Boston, Massachusetts. 4Harvard Medical School, Boston, Massachusetts; Department of Dermatology, Brigham and Women's Hospital, Boston, Massachusetts; Department of Cutaneous Oncology, Dana Farber Cancer Institute, Boston, Massachusetts. Electronic address: cshi@bwh.harvard.edu.

Publication

J Am Acad Dermatol

Summary

Nirogacestat is a first-in-class oral selective gamma secretase inhibitor US Food and Drug Administration-approved in 2023 for desmoid tumors.1,2 Clinical trials report dermatologic adverse events (dAEs) such as “maculopapular rash” (33%), acneiform dermatitis/folliculitis (13%), and hidradenitis (9%),2 with 9/17 (52.9%) of patients in a phase-II trial developing follicular/cystic lesions.3 However, there is currently limited real-world data on nirogacestat-associated dAEs and impact on treatment continuation. This study characterizes nirogacestat-associated dAEs in a real-world multicenter cohort.

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