(Sharecast News) - Specialty pharmaceutical company Diurnal announced on Monday that the US Food and Drug Administration (FDA) has agreed a special protocol assessment (SPA) for 'Chronocort', or modified-release hydrocortisone, for the design, endpoints and statistical analysis approach of a pivotal study of the product, for the treatment for congenital adrenal hyperplasia (CAH).
The AIM-traded firm said the SPA provided an agreement that the phase 3 trial design "adequately addressed" objectives that would support the regulatory submission for drug approval.
It said the pivotal study was titled 'a randomised, double-blind, active-controlled, phase 3 study of Chronocort compared with immediate-release hydrocortisone replacement therapy in participants aged 16-years and over with congenital adrenal hyperplasia', or 'CONnECT', and was expected to start during the second half of 2021.
Study start-up activities had already begun, to enable a timely initiation and recruitment of up to 150 subjects with CAH, who would be treated for a period of 52 weeks.
Diurnal described Chronocort as a preparation of hydrocortisone that had been "specifically designed" for patients with CAH, which is an orphan condition caused by the deficiency of adrenal enzymes, most commonly 21-hydroxylase.
The company holds the relevant orphan drug designation for the use of Chronocort in CAH in the United States.
About two-thirds of CAH patients were estimated to have poor disease control, leading to elevated androgen levels.
The condition is estimated to affect about 16,000 patients in the US.
"We are pleased to have concluded a series of positive discussions with the FDA around the design of our pivotal Chronocort study to support a regulatory submission for marketing approval in the US," said chief executive officer Martin Whitaker.
"During the first half of 2021, the Diurnal team has been working hard to complete the required preparation activities for this trial, and we look forward to starting this study as soon as possible."
At 0829 BST, shares in Diurnal Group were down 0.4% at 62.25p.
11 Jul 2016 06:45