UK specialty drugmaker Diurnal has filed its Alkindi Sprinkle replacement therapy for children with adrenal insufficiency in the US, a group with few treatment options.
Diurnal said the US marketing application follows a meeting with the FDA which said it would be able to file Alkindi (hydrocortisone granules) under the regulatory pathway for generic drugs, which hinges on showing bioequivalence to standard hydrocortisone tablets.
There are estimated to be approximately 4,500 children with adrenal insufficiency under the age of 17 in the US, and Diurnal has also indicated it will ask the FDA to confirm the drug’s orphan status in the US “which requires the company to demonstrate significant clinical benefit for the product compared to existing therapies”.
The US filing comes on schedule and is another boost for Diurnal as it recovers from a tricky period after its other lead product Chronocort failed to meet its primary objective in a pivotal congenital adrenal hyperplasia (CAH) study in 2018.
Chronocort is now set for a marketing application in Europe before year-end, according to remarks made at the company’s recent annual general meeting.
Alkindi has already been approved in Europe and launched in its first markets – including Germany and the UK – and is specifically designed to allow easier dosing in paediatric patients compared to conventional hydrocortisone tablets.
The two halves of the Alkindi capsules are twisted apart and the granules within can poured directly onto the child’s tongue, administered using a spoon, or sprinkled on food.
The US marketing application is particularly important for Diurnal as if approved, Alkindi will become the only licensed treatment specifically designed for use in children with adrenal insufficiency, where there is a significant unmet patient need, says chief executive Martin Whitaker.
He is anticipating approval of Alkindi in the US in “late 2020”, and Diurnal has previously indicated it wants to find a US commercial partner for Alkindi and Chronocort in the first half of next year.
Alkindi’s roll-out and sales growth in Europe are progressing “in line with expectations”, according to Diurnal, which reckons the market opportunity for diseases resulting from cortisol deficiency in the US and Europe is over $3bn.
The drug made its debut in Germany in 2018, marking Diurnal’s first-ever product launch, but suffered a setback after the country’s Institute for Quality and Efficiency in Health Care (IQWiG) concluded it had not been proven to be superior to generic hydrocortisone.
It has since been backed by the Scottish Medicines Consortium (SMC) for reimbursement on the NHS.