Orphan status is granted to products with a limited patient population and is designed to encourage R&D into treatments for rare diseases. It affords 10 years market exclusivity after the grant of a marketing authorisation in Europe. Other benefits include protocol assistance, access to the centralised procedure for the application for marketing authorisation and fee reductions.
The designation is for a modified release hydrocortisone tablet for the treatment of congenital adrenal hyperplasia, a genetic defect of the adrenal glands. The product is being developed by Phoqus Pharmaceuticals, a drug delivery company also based in the UK. Phoqus recently licensed a Diurnal patent that relates to delayed and sustained release therapy, and will apply its proprietary Qtrol technology to the new product.
The tablet formulation developed by Phoqus will allow for the controlled and sustained release of hydrocortisone, a steroid normally released from the adrenal gland, in a manner that mimics the normal physiological circadian rhythm. This marked rhythm, with high levels in the morning and low levels at night, is lost in patients with adrenal insufficiency.
The Qtrol tablet system is made up of a matrix containing the drug that is surrounded on three sides by an insoluble coating, which only allows the drug to be released from one surface. Phoqus says the system can be adapted to provide a range of release profiles, including zero order, increasing release and pulsatile, and the tablets are easy and inexpensive to manufacture.
Diurnal's management believes that the global market for this product could be worth as much as £100 million (€144m). The product is expected to enter human studies in the fourth quarter of this year.
The company said that the product could offer a potential much needed improvement in steroid treatment for patients with congenital adrenal hyperplasia. Current therapies, such as Pfizer's Cortef (hydrocortisone) and Bristol-Myers Squibb's Florinef (fludrocortisone acetate), cannot adequately control the condition, according to the company, which suggests that the new product will provide a more efficacious therapy with improved compliance from patients.
Congenital adrenal hyperplasia was once considered a rare inherited disorder with severe manifestations. Mild congenital adrenal hyperplasia, however, is common, affecting one in 100 to 1,000 persons and frequently eluding diagnosis. Symptoms can include shortened stature, early puberty, severe acne, and virilisation and infertility in females.
Diurnal said it also intends to explore additional opportunities in areas in which steroid levels may be implicated in other diseases such as inflammatory conditions.
Andy Jones, CEO of Phoqus, said: "This is an exciting opportunity to apply Phoqus' innovative Qtrol controlled release technology to develop a product that will benefit patients through simplified dosing regimens and reduced side effects."