UK speciality pharma Diurnal has filed an application in Europe to market Chronocort (modified release hydrocortisone) for patients with the rare condition congenital adrenal hyperplasia (CAH).
The submission is based on analysis of data from a Phase III study, the largest ever interventional clinical trial in CAH, and an open-label safety extension study.
The firm said it expects marketing approval for Chronocort in Europe in the first quarter of next year 2021.
Should Chronocort be approved, patients can start treatment with Alkindi (hydrocortisone granules), Diurnal’s approved paediatric product, transitioning to Chronocort in adolescence and continuing with Chronocort treatment into later life, it noted.
“There is a significant need for new therapies to improve outcomes for adult patients with CAH, which still results in increased morbidity and mortality worldwide,” said Martin Whitaker, the group’s chief executive. “We believe that Chronocort together with our paediatric product Alkindi has the potential to provide new treatment options for patients with CAH.”
CAH is an orphan condition caused by a block in production of the hormone cortisol, a lack of which causes the over-production of male steroid hormones (androgens).
Cortisol deficiency and over-production of androgens can lead to increased mortality, infertility and issues during sexual development, including ambiguous genitalia, precocious puberty and short stature. Sufferers, even if treated, remain at risk of death through an adrenal crisis.
CAH is estimated to affect around 41,000 adult patients in Europe, with over 400,000 in the rest of the world.