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Combination Therapies and Personalized Medicine02:50

Combination Therapies and Personalized Medicine

Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...

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Updated: Jun 19, 2026

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

Maxime Annereau1,2, François-Xavier Legrand3, Maria Virginia Sánchez-Becerra4,5

  • 1Département de Pharmacie Clinique, Gustave Roussy, 94805, Villejuif, France. maxime.annereau@gustaveroussy.fr.

Pharmaceutical Research
|March 26, 2026
PubMed
Summary
This summary is machine-generated.

Hospital pharmacists developed a framework to enable the authorized use of birabresib, a bromodomain and extra-terminal (BET) inhibitor, for treating NUT carcinoma. This approach qualifies discontinued drugs for rare cancer patients.

Keywords:
Capsule compoundingNon-GMP material qualificationQuality & risk-based CMC frameworkStability program

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Area of Science:

  • Pharmaceutical Sciences
  • Oncology
  • Drug Development

Background:

  • NUT carcinoma is an aggressive cancer with no approved systemic treatments.
  • Birabresib (a BET inhibitor) showed early promise but its development was halted, leaving no available GMP-grade drug.
  • Access to birabresib was limited due to the lack of an authorized product.

Purpose of the Study:

  • To describe the pharmaceutical development of birabresib for authorized therapeutic use in France.
  • To establish a hospital-based framework for qualifying a discontinued drug for a rare malignancy.
  • To enable patient access to birabresib under a temporary usage protocol.

Main Methods:

  • A quality- and risk-based Chemistry, Manufacturing, and Controls (CMC) strategy was applied, inspired by ICH Q8-Q10 guidelines.
  • Research-grade birabresib was characterized to establish a reference substance and stability-indicating analytical methods.
  • Compounding of 20-mg capsules was performed under Good Preparation Practice in a hospital pharmacy.

Main Results:

  • Characterization data supported acceptance criteria for drug substance and finished product batches.
  • Finished product controls met pharmacopoeial and ICH requirements.
  • Capsules were entered into a stability program under defined storage conditions.

Conclusions:

  • A risk-proportionate, CMC-focused hospital framework facilitated the first authorized use of birabresib for NUT carcinoma.
  • This model can be adapted for other discontinued small molecules in rare disease access programs.
  • Hospital pharmaceutical development is crucial for enabling access to investigational therapies for ultra-rare diseases.