US FDA Accepts NDA Application for Alkindi

13 February 2020

Diurnal Group plc

("Diurnal" or the "Company")

US FDA Accepts for Review Diurnal's New Drug Application for Alkindi® Sprinkle

Application seeks approval of Alkindi® Sprinkle as a replacement therapy of adrenal insufficiency in infants, children and adolescents

NDA could potentially be approved in Q3 2020

Diurnal Group plc (AIM: DNL), the specialty pharmaceutical company targeting patient needs in chronic endocrine (hormonal) diseases, announces that its New Drug Application (NDA) for Alkindi® (hydrocortisone granules in capsules for opening), to be known in the US as Alkindi® Sprinkle, has been accepted for review by the US Food and Drug Administration (FDA). Diurnal is seeking approval of Alkindi® Sprinkle as a replacement therapy of adrenal insufficiency (AI) in infants, children and adolescents (from birth to

Paediatric AI is a condition characterised by deficiency in cortisol, an essential hormone in regulating growth, metabolism and the response to stress. Paediatric AI has been identified as an orphan disease in the US where there are estimated to over 4,000 sufferers under the age of 17. Untreated, the disease is associated with significant morbidity and increased mortality.

The NDA for Alkindi® Sprinkle was submitted in November 2019 following a positive meeting with the FDA in Q1 2019 which confirmed Diurnal's clinical and regulatory pathway for the product in the US. The PDUFA date set by the FDA, which would be the earliest date at which approval could occur, is 29 September 2020.

Martin Whitaker, CEO of Diurnal, commented: 

"We are pleased to announce that our NDA for Alkindi® Sprinkle in the US has been accepted for review by the FDA. If approved, Alkindi® Sprinkle will provide a major breakthrough in the US as the only licensed treatment specifically designed for use in children with adrenal insufficiency, where there is a significant unmet patient need."

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

Notes to Editors

About Alkindi® Sprinkle (hydrocortisone granules in capsules for opening)

Alkindi® Sprinkle is a patented, oral, immediate-release paediatric formulation of hydrocortisone granules in capsules for opening. Alkindi® Sprinkle comes in dose strengths consistent with age-appropriate dosing in children. This therapeutic approach has the potential to help young patients less than seventeen years of age suffering from diseases due to cortisol deficiency including paediatric AI and congenital adrenal hyperplasia (CAH) in the US. The European Commission has granted a paediatric use marketing authorisation (PUMA) for Alkindi® as replacement therapy of AI in infants, children and adolescents (from birth to

About Paediatric Adrenal Insufficiency

Paediatric AI, including the genetic condition 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).

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: February 2020 Code: Inf US-USA-0005