Paediatric hydrocortisone
Adrenal insufficiency is a rare disorder with a prevalence of approximately 1 in 15 000 people in Europe. Patients do not produce sufficient amounts of the hormone cortisol, an essential component of the physiological response to challenges such as infection, injury or surgery. As a result, any infection, unless treated, may cause adrenal crisis leading to hypotension, circulatory collapse and rapid death. For many years, adrenal insufficiency has been treated with the off-patent drug hydrocortisone, a glucocorticoid hormone used as replacement therapy across Europe. However, effective hydrocortisone replacement is particularly acute among young patients who usually suffer from congenital adrenal hyperplasia genetic cause of adrenal insufficiency. To address this issue, the EU-funded TAIN (Treatment of adrenal insufficiency in neonates - development of a hydrocortisone preparation for the treatment of adrenal insufficiency in neonates, infants and young children) project brought together leaders in drug development, neonatology and paediatric pharmacology. They proposed to develop a new formulation of hydrocortisone suitable for patients of up to six years of age. Using commercially proven technology, the consortium produced the product in four different doses to provide flexibility to the clinicians. To eliminate the bitter taste of hydrocortisone and increase treatment compliance, scientists used taste-masking excipients acceptable for paediatric populations. Formal stability studies were also performed to ensure that the new formulation has a long and practical shelf life for patients and clinicians. Partners employed computational models to minimise testing of the drug in patients. They also obtained approval for their Paediatric Investigation Plan from the EMA, which allowed them to provide substantial evidence on the safety and efficacy of the new hydrocortisone formulation. This culminated with the successful submission of a Paediatric Use Market Authorisation to the EMA. Apart from the novel hydrocortisone formulation, TAIN succeeded in raising awareness of the unmet needs in paediatric adrenal insufficiency patients. The new pharmaceutical product is expected to benefit children and their families across Europe.
Keywords
Hydrocortisone, adrenal insufficiency, EMA, TAIN, Paediatric Use Market Authorisation
