US FDA confirms the current clinical and regulatory path for Alkindi: Diurnal on track to submit NDA in Q4 2019
Diurnal Group plc (AIM: DNL), the specialty pharmaceutical company targeting patient needs in chronic endocrine (hormonal) diseases, announces that following a positive meeting with the US Food and Drug Administration (FDA), the Company intends to submit a New Drug Application (NDA) for Alkindi® (hydrocortisone granules in capsules for opening) as planned in Q4 2019, with final acceptance anticipated by the Company in 2020.
During the meeting, the FDA confirmed the current clinical and regulatory path for Alkindi® as a replacement therapy for paediatric adrenal insufficiency (AI) in the US. This follows completion of a development programme for Alkindi®, including a study to demonstrate bioequivalence with the US reference product, as announced on 17 October 2018.
Martin Whitaker, Chief Executive Officer of Diurnal, commented:
“We are pleased with the outcome of our meeting with the FDA following completion of the development programme for Alkindi® in the US. If accepted by the FDA, Alkindi® will be the first licensed treatment in the US specifically designed for use in children with adrenal insufficiency, where there is a significant unmet patient need. Following this positive development, we are now progressing discussions with potential partners for Alkindi® in the US who are able to optimise market access for this important product.”
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 be approximately 4,500 sufferers under the age of 16. Untreated, the disease is associated with significant morbidity and increased mortality. Alkindi® has the potential to be the first pharmaceutically defined dose and consistent formulation of hydrocortisone (the synthetic version of cortisol) designed specifically for paediatric patients.
In the US, Diurnal holds Orphan Drug Designation for Alkindi® for paediatric AI, which affords seven years’ market exclusivity post New Drug Application approval. A robust, granted, patent portfolio provides in-market protection for Alkindi® to 2034, which significantly bolsters the Company’s exclusivity position.
Until the NDA has been accepted for filing and fully reviewed by FDA final acceptance cannot be assumed.
This announcement contains inside information for the purposes of Article 7 of Regulation (EU) 596/2014 (MAR).
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
Panmure Gordon (UK) Limited (Nominated Adviser and Joint Broker) +44 (0) 20 7886 2500
Corporate Finance: Freddy Crossley, Emma Earl
Corporate Broking: James Stearns
Cantor Fitzgerald Europe (Joint Broker) +44 (0) 20 7894 7000
Corporate Finance: Phil Davies, Will Goode, Michael Boot
Healthcare Equity Sales: Andrew Keith
FTI Consulting +44 (0)20 3727 1000
Victoria Foster Mitchell
Notes to Editors
About Alkindi® (hydrocortisone granules in capsules for opening)
Alkindi® is the first preparation of hydrocortisone specifically designed for use in children suffering from paediatric adrenal insufficiency (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 congenital adrenal hyperplasia (CAH). AI requires life-long treatment and Diurnal’s novel approach to product development has the potential to significantly improve these young patients’ lives. 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 <18 years old) in Europe.
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: March 2019 Code: Inf US-GB-0004
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