Posted on Tuesday, 13/Feb/2018 Posted by michelle

First licensed treatment in Europe specifically designed for use in children with adrenal insufficiency

Diurnal’s first product to receive marketing authorisation

Market launch anticipated in Q2 2018

Diurnal Group plc (AIM: DNL), the specialty pharmaceutical company targeting patient needs in chronic endocrine (hormonal) diseases, is pleased to announce that the European Commission (EC) has granted a paediatric use marketing authorisation (PUMA) for Alkindi® (development programme name: Infacort®; hydrocortisone granules in capsules for opening) as replacement therapy of adrenal insufficiency in infants, children and adolescents (from birth to <18 years old), following the positive opinion issued by the European Medicines Agency (EMA) in December 2017.

Alkindi® is specifically designed to provide the first regulated, consistent preparation of hydrocortisone (the synthetic version of cortisol) to ensure efficacy and safety and ease of use for children suffering from AI. Having adopted the EMA decision, the EC marketing approval under a PUMA affords 10 years’ data and market exclusivity for Alkindi® in Europe.

Given the specialist prescribing base, and to retain the full commercial value of the product, Diurnal will commercialise Alkindi® itself in the major European markets. Diurnal has a detailed commercial plan in place for the anticipated first launch of Alkindi® in Q2 2018.

Martin Whitaker, Chief Executive Officer of Diurnal, commented:
“We are delighted to receive European approval for Alkindi®, a significant milestone for Diurnal as our first marketing authorisation. This approval is a major breakthrough for paediatric patients with adrenal insufficiency, with Alkindi® being the first licensed treatment in Europe specifically designed for use in these children. Paediatric AI is currently treated by compounding adult preparations of hydrocortisone, which has been approved for more than 50 years. We have a detailed commercial plan in place to bring this important new treatment to paediatric patients and address the current unmet medical need. We anticipate the first launch of Alkindi® in Q2 2018.”

The marketing authorisation approval is based upon 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 market approval now extends the product’s use to include paediatric patients up to 18 years of age.

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 where the need for Alkindi® is greatest. Untreated, the disease is associated with significant morbidity and increased mortality.

For further information, please visit www.diurnal.co.uk or contact:

Diurnal Group plc +44 (0)20 3727 1000
Martin Whitaker, Chief Executive Officer 
Richard Bungay, Chief Financial Officer 

Numis Securities Ltd (Nominated Adviser) +44 (0)20 7260 1000
Nominated Adviser: Michael Meade, Paul Gillam, Freddie Barnfield 
Corporate Broking: James Black 

Panmure Gordon (UK) Limited (Joint Broker) +44 (0) 20 7886 2500
Corporate Finance: Freddy Crossley 
Corporate Broking: Tom Salvesen 

FTI Consulting +44 (0)20 3727 1000
Simon Conway 
Victoria Foster Mitchell

Notes to Editors

About Alkindi®
Alkindi® represents the first licensed preparation of hydrocortisone specifically designed for use in children suffering from paediatric AI in Europe. Alkindi® is a patented, oral, immediate-release paediatric formulation of hydrocortisone granules in capsules for opening that allows for age-appropriate dosing in children. This therapeutic approach has the potential to help young patients less than eighteen years of age in Europe suffering from diseases due to cortisol deficiency including paediatric AI and CAH. AI requires life-long treatment and Diurnal’s novel approach to product development has the potential to significantly improve these young patients’ lives.

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