DNL.L Regulatory News (DNL)

15 December 2017

Diurnal Group plc

("Diurnal" or the "Company")

CHMP issues positive opinion for Alkindi® (Infacort®) for the treatment of paediatric adrenal insufficiency in Europe

 Positive opinion for approval includes paediatric patients up to 18 years of age

Alkindi® will be Diurnal's first product to receive market authorisation; market launch anticipated in Q2 2018

Diurnal Group plc (AIM: DNL), the specialty pharmaceutical company targeting patient needs in chronic endocrine (hormonal) diseases, announces that the Committee for Medicinal Products for Human Use (CHMP), an advisory committee of the European Medicine Agency (EMA), has issued a positive opinion to the European Commission recommending Alkindi® (development programme name: Infacort®; hydrocortisone granules in capsules for opening) as replacement therapy for paediatric adrenal insufficiency (AI). In accordance with the 60-day timeline following the adoption of the positive opinion by the CHMP, the final decision on the paediatric use marketing authorisation (PUMA) from the European Commission is anticipated in February 2018. 

The positive opinion from the CHMP is based on review of data from the Company's pivotal open-label Phase III clinical trial conducted in 24 subjects before their sixth birthday, requiring replacement therapy for adrenal insufficiency due to congenital adrenal hyperplasia, primary adrenal failure or hypopituitarism. The study successfully met its primary endpoint and no serious adverse events were reported. Based on these data, and a comprehensive dossier from Diurnal, the CHMP has recommended the product's use to include paediatric patients up to 18 years of age.

Reflecting a concentrated prescribing base, and to retain the full value of the product, Diurnal intends to commercialise Alkindi® itself in the major European markets, focusing its marketing efforts initially on patients aged 0-6 years where the unmet need is highest. Diurnal has made significant progress during 2017 in establishing its European commercial operations to prepare for the anticipated launch of Alkindi® in Q2 2018.

Paediatric AI, including the related genetic condition Congenital Adrenal Hyperplasia (CAH), is a condition characterised by deficiency in cortisol, an essential hormone in regulating metabolism and the response to stress. Paediatric AI has been identified as a rare disease in Europe where there are estimated to be approximately 4,000 sufferers under the age of six. Untreated, the disease is associated with significant morbidity and increased mortality.

AI in children is currently treated by compounding hydrocortisone or crushing/splitting tablets of adult preparations of hydrocortisone (the synthetic version of cortisol) as there is no licensed formulation available specifically designed for children. Alkindi® is specifically designed to provide the first regulated, consistent preparation of hydrocortisone to ensure efficacy and safety and ease of use for children suffering from AI, including CAH.

Dr Oliver Blankenstein of Charité-Universitätsmedizin Berlin and co-investigator in the Phase III clinical trial commented:

"I am very pleased that this medicine, specifically designed to treat children with adrenal insufficiency, may soon be available to patients in Europe, following the issue of a positive opinion for approval by the CHMP. I believe Alkindi® is a major breakthrough in addressing the significant unmet medical need in the paediatric population."

Martin Whitaker, CEO of Diurnal, added:

"We are delighted that the CHMP recognises Alkindi® as a treatment for paediatric patients with paediatric adrenal insufficiency. If approved by the European Commission, Alkindi® will be the first licensed treatment in Europe specifically designed for use in children with adrenal insufficiency. We are on track with establishing our European infrastructure to directly commercialise Alkindi® in key territories, subject to receiving paediatric use marketing authorisation for this important new treatment, which is anticipated in February 2018."

Notes to Editors

About Diurnal Group plc

Founded in 2004, Diurnal is a UK-based specialty pharma company developing high quality products for the global market for the life-long treatment of chronic endocrine conditions, including Congenital Adrenal Hyperplasia and Adrenal Insufficiency. Its expertise and innovative research activities focus on circadian-based endocrinology to yield novel product candidates in the rare and chronic endocrine disease arena.

For further information about Diurnal, please visit www.diurnal.co.uk