Hospital-Compounded Birabresib Capsules for NUT Carcinoma: A Quality- and Risk-Based CMC Strategy

20260 citationsJournal Articlehybrid Open Access

Authors

Maxime Annereau · Inserm

François‐Xavier Legrand · Centre National de la Recherche Scientifique

Maria Virginia Sanchez-Becerra · Inserm

Stéphanie Ramos · Centre National de la Recherche Scientifique

Françoise Duperray · Agence Nationale de Sécurité du Médicament et des Produits de Santé

Lucas Denis · Centre National de la Recherche Scientifique

Christina Sizun

Abstract

This risk-proportionate, CMC-oriented hospital framework enabled the first authorised therapeutic use of birabresib in NUT carcinoma and may be extended to other discontinued small molecules used in regulated access programmes for ultra-rare diseases.

Topics & Keywords

Protein Degradation and Inhibitors PI3K/AKT/mTOR signaling in cancer Sirtuins and Resveratrol in Medicine

UN Sustainable Development Goals

Zero hunger

Publication Details

Published in: Pharmaceutical Research

DOI: 10.1007/s11095-026-04081-9

Field-Weighted Citation Impact: 0.00