DNL.L Regulatory News (DNL)

8 October 2018

Diurnal Group plc

("Diurnal" or the "Company")

Scottish Medicines Consortium approves the use of Alkindi® as first-line treatment for paediatric adrenal insufficiency

Diurnal Group plc (AIM: DNL), the specialty pharmaceutical company targeting patient needs in chronic endocrine (hormonal) diseases, is pleased to announce that the Scottish Medicines Consortium (SMC) has today approved the routine use of Alkindi® (hydrocortisone granules in capsules for opening) as the first-line treatment of young children with adrenal insufficiency aged from birth to less than six years of age for whom hydrocortisone must be individually prepared by manipulation such as by compounding (or crushing) or by production of special solutions in order to produce age-appropriate doses, or hydrocortisone given as off-label buccal tablets.

The decision by the SMC means that children in Scotland with this rare condition, including those with congenital adrenal hyperplasia (CAH), can now be treated with accurate dosing of licensed hydrocortisone medicine and without the need to crush or dissolve tablets. Importantly, the SMC recommended Alkindi® to be cost-effective for use in children less than six years old, which are the most vulnerable adrenal insufficiency (AI) patients and where the unmet need is highest.

Given the concentrated prescribing base, and to retain the full value of the product, Diurnal intends to commercialise Alkindi® in the UK and other major European markets. Diurnal has established the commercial infrastructure required to support a successful launch of Alkindi®, focusing its marketing efforts initially on these 0-6 year old patients. 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 following the grant of a paediatric use marketing authorisation (PUMA) by the European Commission in February 2018. Alkindi® was launched in Germany in May 2018 and in the UK in September 2018.

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

Prof Faisal Ahmed, Clinical Endocrinologist, University of Glasgow, added:

"We welcome this decision by the SMC. Administering a hydrocortisone dose titrated to children's needs is essential. Previously, licensed hydrocortisone was only available in adult dose sizes, making accurate dosing for children difficult. Alkindi® provides the ability to prescribe and administer small doses which is particularly important for young children. We look forward to finally using a preparation which is specifically designed for this group of patients."

Martin Whitaker, Chief Executive Officer of Diurnal, commented: 

"We are delighted that the SMC has recommended, in line with our expectations, the first-line use of Alkindi® in paediatric adrenal insufficiency in children under six years of age. Diurnal believes the use of this product will improve the lives of patients with this rare disease, and their families, by providing access to the first formulation of hydrocortisone specifically designed for paediatric dosing. Diurnal hopes that health providers in other parts of the UK, and across Europe, will follow a similar positive decision to enable parents to provide accurate dosing of life-saving medicine for their children."

The full text of the SMC decision can be found at: https://www.scottishmedicines.org.uk/medicines-advice/hydrocortisone-alkindi-fullsub-smc2088/

This announcement contains inside information for the purposes of Article 7 of Regulation (EU) 596/2014 (MAR).

Notes to Editors

About The Scottish Medicines Consortium

The SMC is part of Healthcare Improvement Scotland, the national healthcare improvement organisation for Scotland. The SMC reviews new medicines that have received a licence from the Medicines and Healthcare products Regulatory Agency (MHRA - the licensing body for the UK) or the European Medicines Agency (EMA - the licensing body for the European Union). The SMC also reviews new formulations of, and new ways to use, established medicines. Before a medicine can be prescribed routinely in Scotland, it has to be accepted for use by SMC. SMC advice helps the health service plan for the quick, uniform introduction of beneficial treatments across NHSScotland. 

About Alkindi® (hydrocortisone granules in capsules for opening)

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 accurate 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 ® in Europe.

About Paediatric Adrenal Insufficiency

Paediatric Adrenal insufficiency (AI), including the 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. 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 hydrocortisone (the synthetic version of cortisol) as there was no licensed formulation available specifically designed for children.

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

Date of Preparation: October 2018 Code: Inf EU-EU-0085