Diurnal Limited (“Diurnal”), one of Biofusion plc’s portfolio companies, announces that the European Commission has granted Orphan Medicinal Product Designation to Chronocort™ for the treatment of adrenal insufficiency (“AI”). This is the second indication for which ChronocortTM has received Orphan Medicinal Product Designation from the European Commission. Orphan Medicinal Product Designation was originally granted in respect of the genetic disorder congenital adrenal hyperplasia (“CAH”) in 2005.
The EU Orphan Medicinal Product Designation is intended to promote the development of drugs that may provide significant benefit to patients suffering from rare diseases identified as serious or life-threatening. Under EMEA guidelines, Orphan Medicinal Product Designation provides the sponsor access to free protocol assistance to aid the development of clinical trials, waivers of fees relating to the marketing approval process, centralised registration procedure and ten years of marketing exclusivity once the product is approved for the designated indication in the European Union. Early discussions are now underway with the US Food and Drug Administration (“FDA”) to seek equivalent orphan drug designations in the US.
Chronocort™ is being developed by Phoqus Pharmaceuticals Limited (“Phoqus”), a drug delivery company based in Kent, England. This follows the exclusive licencing by Phoqus of a Diurnal patent that relates to delayed and sustained release therapy. Chronocort™ is a modified release tablet containing hydrocortisone for both AI, the failure of the adrenal glands to produce sufficient steroid hormones, and congenital CAH. CAH is a serious genetic disorder caused by the deficiency of an enzyme responsible for cortisol production.
ChronocortTM is designed to provide a new form of corticosteroid hormone replacement therapy by releasing hydrocortisone in a manner that will enable doctors to achieve a daily cycle (circadian rhythm) of cortisol levels in patients that closely matches that of the normal population. This in turn should improve disease symptom control and may also increase the accuracy of the disease treatment and monitoring regimen, potentially reducing the incidence of over or under exposure to steroids.
Planning is underway for the full clinical development programme which includes pivotal pre-registration studies in both CAH patients and patients suffering from AI that are scheduled to commence in the latter half of 2007 in both the EU and the US, subject to regulatory approval. Chronocort™ is currently under evaluation in healthy volunteer studies to confirm its pharmacokinetic profile.
In the EU and US combined there are estimated to be between 78,000 and 206,000 patients with acquired AI (due to conditions such as Addison’s Disease and hypopituitarism) and a further 44,000 to 53,000 patients with the genetic disorder CAH.
Phoqus' CEO, Dr. Richard Mason, commented:
“Adrenal insufficiency is a disease of significant unmet therapeutic need and results in patients having a quality of life similar to chronic congestive heart failure. Subject to regulatory approval, we are planning to commence pivotal pre-registration clinical trials for both adrenal insufficiency and congenital adrenal hyperplasia in the second half of this year, and the orphan drug designation granted by the EMEA will be of great benefit to our clinical programme. Based on reasonable assumptions, we believe that Chronocort has the potential to generate peak annual sales in excess of $200m from use in these two orphan indications.”