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

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.