LONDON (Alliance News) - Diurnal Group PLC said Monday the Scottish Medicines Consortium approved the routine use of Alkindi, a first-line treatment of young children with adrenal insufficiency aged from birth to less than six.
The pharmaceutical company said the decision means children in Scotland with the rare condition, including those with congenital adrenal hyperplasia, can now be treated with accurate dosing of licensed hydrocortisone medicine.
Diurnal estimates that there are approximately 4,000 patients in Europe under the age of six requiring replacement therapy for adrenal insufficiency due to congenital adrenal hyperplasia, primary adrenal failure or hypopituitarism.
Diurnal intends to commercialise Alkindi - hydrocortisone granules in capsules - in the UK and other European markets. The company is currently in discussion with "various health authorities" across Europe.
Chief Executive Officer Martin Whitaker said: "Diurnal believes the use of this product will improve the lives of patients with this rare disease, and their families, by providing access to the first formulation of hydrocortisone specifically designed for paediatric dosing.
"Diurnal hopes that health providers in other parts of the UK, and across Europe, will follow a similar positive decision to enable parents to provide accurate dosing of life-saving medicine for their children."
Shares in Diurnal more than halved Monday, trading down 61% to 42.10 pence each as the company's phase-three European trial of Chronocort did not meet its primary objective.
Intellectual property business developer IP Group PLC, which holds a 44% stake in Diurnal, made a separate announcement noting the result.
Chronocort is intended to treat congenital adrenal hyperplasia, an orphan condition in which patients have a block in cortisol production. This lack of cortisol causes the over-production of male hormones, and can lead to infertility and increased mortality.
The goal of the phase three trial was to "demonstrate the superiority of Chronocort to conventional therapy", but was not met as both Chronocort and conventional therapy proved equivalent.
Diurnal said that while the primary objective was not met, the trial did show that Chronocort achieved control on a lower overall dose with fewer patients requiring rescue therapy.
