Diurnal Group plc, the specialty pharmaceutical company targeting patient needs in chronic endocrine (hormonal) diseases, announced that the European Commission (EC) has granted a paediatric use marketing authorisation (PUMA) for Alkindi (development programme name: Infacort; hydrocortisone granules in capsules for opening) as replacement therapy of adrenal insufficiency in infants, children and adolescents (from birth to <18 years old), following the positive opinion issued by the European Medicines Agency (EMA) in December 2017.
Alkindi is specifically designed to provide the first regulated, consistent preparation of hydrocortisone (the synthetic version of cortisol) to ensure efficacy and safety and ease of use for children suffering from AI. Having adopted the EMA decision, the EC marketing approval under a PUMA affords 10 years’ data and market exclusivity for Alkindi in Europe.
Given the specialist prescribing base, and to retain the full commercial value of the product, Diurnal will commercialise Alkindi itself in the major European markets. Diurnal has a detailed commercial plan in place for the anticipated first launch of Alkindi in Q2 2018.
Martin Whitaker, Chief Executive Officer of Diurnal, commented: “We are delighted to receive European approval for Alkindi, a significant milestone for Diurnal as our first marketing authorisation. This approval is a major breakthrough for paediatric patients with adrenal insufficiency, with Alkindi being the first licensed treatment in Europe specifically designed for use in these children. Paediatric AI is currently treated by compounding adult preparations of hydrocortisone, which has been approved for more than 50 years. We have a detailed commercial plan in place to bring this important new treatment to paediatric patients and address the current unmet medical need. We anticipate the first launch of Alkindi in Q2 2018.”
The marketing authorisation approval is based upon review of data from the Company’s pivotal open-label Phase III clinical trial conducted in 24 subjects before their sixth birthday, requiring replacement therapy for adrenal insufficiency due to congenital adrenal hyperplasia, primary adrenal failure or hypopituitarism. The study successfully met its primary endpoint and no serious adverse events were reported. Based on these data, and a comprehensive dossier from Diurnal, the market approval now extends the product’s use to include paediatric patients up to 18 years of age.
Paediatric AI, including the related genetic condition congenital adrenal hyperplasia (CAH), is a condition characterised by deficiency in cortisol, an essential hormone in regulating metabolism and the response to stress. Paediatric AI has been identified as a rare disease in Europe where there are estimated to be approximately 4,000 sufferers under the age of six where the need for Alkindi is greatest. Untreated, the disease is associated with significant morbidity and increased mortality.