IP Group plc (LON: IPO), the developer of intellectual property-based businesses, notes the announcement by its portfolio company Diurnal Group plcthat, following a positive meeting with the European Medicines Agency (EMA) in March 2019, it has received formal Scientific Advice from the EMA confirming the current clinical and regulatory path for Chronocort® as a treatment for adults with congenital adrenal hyperplasia (CAH).
Consequently, Diurnal intends to submit a Marketing Authorisation Application (MAA) for Chronocort® (modified release hydrocortisone) in Q4 2019 based upon the existing clinical data, including data to support Orphan Drug Status in the treatment of CAH. This positive outcome follows the submission of a regulatory package requesting Scientific Advice to the EMA for Chronocort® based on detailed analysis of data from its Phase 3 study, the largest ever clinical trial programme in CAH, and the open-label safety extension study.
IP Group currently holds a direct undiluted beneficial stake of 43.3% in Diurnal, a specialty pharmaceutical company targeting patient needs in chronic endocrine (hormonal) diseases.
Dr Sam Williams, IP Group Managing Partner, Life Sciences, said: “This positive development means that the EMA has not requested a further clinical study of Chronocort, despite the recent Phase 3 not meeting its primary endpoint, and is consistent with Diurnal’s intention to file its marketing application this year based on the existing data. This supports IP Group’s view that the chances of approval of Chronocort in Europe are very good and, moreover, that there remains the potential for Orphan status, which would give Chronocort significant advantages over existing treatments for CAH.”
CAH is an orphan condition caused by a block in cortisol production, an essential adrenal steroid hormone required for healthy life. A lack of cortisol in turn causes the over-production of male steroid hormones (androgens). Cortisol deficiency and over-production of androgens can lead to increased mortality, infertility and severe development defects, including ambiguous genitalia, precocious puberty and short stature. Sufferers, even if treated, remain at risk of death through an adrenal crisis. The condition is estimated to affect a total of approximately 47,000 patients in Europe, with over 400,000 in the rest of the world.