Hydrocortisone Aguettant
Application withdrawn
The application for this medicine has been withdrawn
Medicine
Human
Application withdrawn
- Application under evaluation
- Withdrawal of application
Overview
Laboratoire Aguettant withdrew its application for a marketing authorisation of Hydrocortisone Aguettant for the prevention of bronchopulmonary dysplasia in preterm infants.
The company withdrew the application on 18 September 2025.
Hydrocortisone Aguettant was developed as a medicine to prevent bronchopulmonary dysplasia in preterm (premature) infants between 24 and 28 weeks’ postmenstrual age (after the mother’s last menstruation). Bronchopulmonary dysplasia is a chronic (long-term) lung disease affecting preterm infants who have been on prolonged mechanical ventilation (a machine that supplies oxygen to help with breathing). Preterm infants may have breathing difficulties because their lungs are immature and therefore may need mechanical ventilation to force oxygen into their lungs. However, this can cause inflammation and injury to the lungs, leading to long‑term breathing problems.
Hydrocortisone Aguettant contains the active substance hydrocortisone and was to be available as a powder and solvent to be made up into a solution for injection into a vein.
Hydrocortisone Aguettant was developed as a hybrid medicine. This means that Hydrocortisone Aguettant is similar to a reference medicine containing the same active substance, but there are certain differences between the two. In this application, Hydrocortisone Aguettant was intended for use at a different concentration and for a different indication than that of the reference medicine, Hydrocortisone Upjohn.
Preterm infants born before 28 weeks’ postmenstrual age may produce low levels of a hormone called cortisol which helps to control inflammation. As a result, their bodies are less capable of controlling the inflammation in their lungs caused by prolonged mechanical ventilation. The active substance in Hydrocortisone Aguettant, hydrocortisone, is a synthetic (man-made) form of cortisol which was expected to help to restore the hormone levels, thereby reducing inflammation in the lungs.
The company presented results from published literature, including a study involving 523 infants born between 24 and 28 weeks of postmenstrual age, which compared treatment with Hydrocortisone Aguettant with placebo (a dummy treatment) during the first 10 days of life. The study looked at how many infants were alive and did not have bronchopulmonary dysplasia at 36 weeks’ postmenstrual age.
The application was withdrawn after the European Medicines Agency had evaluated the information from the company and prepared questions for the company. After the Agency had assessed the company’s responses to the last round of questions, there were still some unresolved issues.
Based on the review of the data and the company’s response to the Agency’s questions, at the time of the withdrawal, the Agency had concerns and its provisional opinion was that Hydrocortisone Aguettant could not have been authorised for the prevention of bronchopulmonary dysplasia.
The Agency had uncertainties about the effectiveness data in terms of the number of infants alive and without bronchopulmonary dysplasia at 36 weeks. Due to limitations in how the study and the analyses had been carried out, the Agency questioned the robustness of the data and the relevance of the effect for patients. Moreover, additional data did not provide any supportive information, nor were there any data on the medicine’s effects in the long term. Lastly, the study had been conducted 10 years ago and there was limited support from other available sources. It was therefore uncertain whether the results would be relevant for neonatal (newborn) care today, as the standard of care has developed since the study had been conducted.
In terms of safety, there were concerns about an increase in certain serious side effects seen during treatment with Hydrocortisone Aguettant, particularly in infants born before 26 weeks’ postmenstrual age and in those who had adequate levels of cortisol before treatment. These include sepsis (blood poisoning), serious gastrointestinal disorders (disorders affecting the stomach and intestines) and bleeding in the fluid-filled areas (ventricles) inside the brain (intraventricular haemorrhage).
There were also some concerns relating to the documentation of certain quality aspects.
Therefore, at the time of the withdrawal and based on the submitted data, the Agency’s opinion was that the benefits of Hydrocortisone Aguettant did not outweigh its risks.
In its letter notifying the Agency of the withdrawal of the application, the company stated that the withdrawal was based on feedback from EMA that, based on the data provided, the Agency could not conclude that the benefits of the medicine outweighed its risks.
The company informed the Agency that there are no consequences for patients in clinical trials using Hydrocortisone Aguettant.
If your infant is in a clinical trial and you need more information about your infant’s treatment, speak with your clinical trial doctor.
Key facts
- Name of medicine
- Hydrocortisone Aguettant
- Active substance
- hydrocortisone
- Therapeutic area (MeSH)
- Bronchopulmonary Dysplasia
- EMA product number
- EMEA/H/C005201
- Marketing authorisation applicant
- Laboratoire Aguettant
- Withdrawal of application
- 18/09/2025
All documents
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