What question did this study set out to answer?

The aim is to establish a quality-focused framework for the therapeutic use of birabresib in treating NUT carcinoma.

March 28, 2026Pharmaceutical Research0 citationsOpen Access

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

MAMaxime Annereau FLFrançois‐Xavier Legrand MSMaria Virginia Sanchez-Becerra

Key Points

The aim is to establish a quality-focused framework for the therapeutic use of birabresib in treating NUT carcinoma.
Developed a risk-proportionate CMC-oriented framework.
Enabled the first authorized therapeutic use of birabresib.
Designed to apply to other small molecules in regulated access programs.
Successfully authorized birabresib for NUT carcinoma.
Potential to extend the framework to other ultra-rare diseases.

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.

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Cite This Study

Annereau et al. (Wed,) studied this question.

synapsesocial.com/papers/69c770c08bbfbc51511e0c24 https://doi.org/https://doi.org/10.1007/s11095-026-04081-9

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