EffRx: Swiss marketing authorization for paediatric adrenocortical insufficiency treatment

EffRx Pharmaceuticals SA, a commercial-stage company that commercializes niche and orphan medicines in Switzerland and Europe, today announced that Swissmedic has approved Alkindi for the treatment of paediatric adrenocortical insufficiency (AI).

Alkindi, developed by Diurnal Group plc, is the first preparation of hydrocortisone specifically designed for use in children suffering from paediatric adrenocortical insufficiency (AI). Alkindi is a patented, oral, immediate-release paediatric formulation of hydrocortisone granules in capsules for opening that allows for accurate age-appropriate dosing in children. This therapeutic approach has the potential to help young patients less than eighteen years of age suffering from paediatric AI and the related condition congenital adrenal hyperplasia (CAH).

Alkindi is approved and marketed in the European Union and is the first preparation of hydrocortisone specifically designed for use in children suffering from AI. On September 29th, 2020 the US Food and Drug Administration (FDA) has also approved Alkindi for AI. This new treatment approach is expected to be available on the Swiss market by H1 2022. In Switzerland, there are approximately 200 patients suffering from paediatric AI.

The marketing authorisation approval in Switzerland is based upon a pivotal open-label Phase III clinical trial conducted in 24 children, requiring replacement therapy for adrenal insufficiency due to congenital adrenal hyperplasia or hypopituitarism. The study successfully met its primary endpoint and no serious adverse events were reported.

“We are delighted to have received Swissmedic approval for Alkindi. This approval is a major breakthrough for paediatric patients with adrenal insufficiency, with Alkindi being the first licensed treatment in Switzerland specifically designed for use in infants, children and adolescents (from birth to less than 18 years old)” commented Lorraine Zakin, Senior Director Medical Affairs at EffRx Pharmaceuticals.

Paediatric AI and the related genetic condition CAH is a condition characterised by a deficiency in cortisol, an essential hormone in regulating metabolism and the response to stress. The primary symptoms of AI are chronic fatigue and patients are at risk of adrenal crisis and death if they do not have adequate cortisol replacement. AI is either primary or secondary, with primary AI resulting from diseases intrinsic to the adrenal gland and secondary AI resulting from pituitary diseases where there is a failure of stimulation of the adrenal by the pituitary of the signalling hormone ACTH (adrenocorticotropic hormone).