Posted on Tuesday, 15/May/2018 Posted by michelle

First Europe-wide licensed treatment specifically designed for younger children with adrenal insufficiency, an underserved population

First country to launch Alkindi®

First Diurnal product to be commercialised

Diurnal Group plc (AIM: DNL), the specialty pharmaceutical company targeting patient needs in chronic endocrine (hormonal) diseases, announces the launch of Alkindi® (hydrocortisone granules in capsules for opening) in Germany as replacement therapy of adrenal insufficiency (AI) in infants, children and adolescents (from birth to <18 years old), following the grant of European marketing authorisation in February 2018.

Alkindi® pricing, in line with the Company’s expectations, has been published today in the LAUER-TAXE® (the reference for all German pharmacies, insurers and wholesalers), following submission of a pricing and reimbursement dossier to The German Federal Committee Gemeinsame Bundesausschuss (G-BA).

As part of the pan-European commercialisation programme for Alkindi®, Diurnal is currently in discussion with various health authorities across Europe to ensure timely launches in other major European countries.

Given the concentrated prescribing base, and to retain the full value of the product, Diurnal intends to commercialise Alkindi® itself in major European markets, focusing its marketing efforts initially on patients aged 0-6 years where the unmet need is highest. Diurnal has established the commercial infrastructure required to support a successful launch of Alkindi®.

Diurnal estimates that there are approximately 4,000 patients in Europe under the age of six requiring replacement therapy for AI due to congenital adrenal hyperplasia, primary adrenal failure or hypopituitarism.

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, is available in Germany. Lack of specific paediatric medicines is a significant unmet medical need in the paediatric population. Alkindi® is the result of an excellent collaboration between the pharmaceutical industry and academia to address the shortfall in paediatric specific medicines.”

The European launch of Alkindi® is underpinned by the data from the Company’s pivotal open-label Phase III clinical trial conducted in 24 subjects before their sixth birthday, requiring replacement therapy for AI due to congenital adrenal hyperplasia (CAH), primary adrenal failure or hypopituitarism. This study, which was conducted at the Charite-Universitatsmedizin hospital in Berlin, successfully met its primary endpoint and no serious adverse events were reported.

Paediatric AI, including the related genetic condition CAH, is a condition characterised by deficiency in cortisol, an essential hormone in regulating metabolism and the response to stress. 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 (1,2,3). 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. 

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, Emma Earl
Corporate Broking: James Stearns

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

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

About Adrenal Insufficiency
Adrenal insufficiency (AI) is a condition characterised by deficiency in cortisol, an essential hormone in regulating metabolism and the response to stress. The primary symptoms of AI are chronic fatigue and patients are at risk of adrenal crisis and death if they do not have adequate cortisol replacement. AI is either primary or secondary, with primary AI resulting from diseases intrinsic to the adrenal gland and secondary AI resulting from pituitary diseases where there is a failure of stimulation of the adrenal by the pituitary of the signalling hormone ACTH (adrenocorticotropic hormone). In Europe, AI has been identified as a rare condition, where there are estimated to be approximately 4,000 sufferers younger than the age of six, where the need for an effective replacement therapy is greatest. Prior to the European approval of Diurnal’s product, Alkindi®, paediatric AI was treated by compounding hydrocortisone or crushing/splitting tablets of adult preparations of hydrocortisone (the synthetic version of cortisol) as there was no licensed formulation available specifically designed for children.

About Alkindi®
Alkindi® is the first preparation of hydrocortisone specifically designed for use in children suffering from paediatric AI. 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 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. On 9 February 2018, the European Commission granted a paediatric use marketing authorisation (PUMA) for Alkindi® as replacement therapy of AI in infants, children and adolescents (from birth to <18 years old), following the positive opinion issued by the European Medicines Agency in December 2017. The PUMA affords 10 years market and data exclusivity for Alkindi® in Europe.

References:

1. Saimbi et al. Comparison of hydrocortisone 10 mg tablets: tablet hardness optimised for adult use has negative consequences for paediatric use. Arch Dis Child. 2016 Sep;101(9):e2.

2. Madathilethu et al. Content uniformity of quartered hydrocortisone tablets in comparison with mini-tablets for paediatric dosing. BMJ Paediatr Open. 2018 Jan 29;2(1):e000198

3. Webb et al. Hydrocortisone tablets: human factors in manipulation and their impact on dosing accuracy.  Endocrine Abstracts (2017) Vol. 51 OC 8.1