Immediate-release granule formulation of hydrocortisone, Alkindi®, for treatment of paediatric adrenal insufficiency (Infacort development programme)

ABSTRACT

Introduction: Treatment of paediatric patients with adrenal insufficiency is challenging due to the lack of appropriate glucocorticoid preparations for children, and the use of either pharmacy- or parent-compounded hydrocortisone tablets. Alkindi (hydrocortisone granules in capsules for opening) is a new therapeutic option for paediatric adrenal insufficiency.

Areas Covered: Drawbacks of current therapy and formulation and clinical trial programme for Alkindi.

Expert Commentary: Compounding hydrocortisone has multiple issues including inconsistent dosing with under and over treatment and practical problems for parents who compound the drug themselves or travel long distances to a compounding pharmacy and the cost of compounding by the pharmacy. Alkindi® is a novel paediatric formulation of immediate release hydrocortisone licensed for use in paediatric adrenal insufficiency. Alkindi® is formulated to address the needs of neonates, infants and young children, being available at appropriate paediatric doses of 0.5, 1.0, 2.0 and 5.0 mg, is multiparticulate, allowing either direct oral dosing or dosing mixed with food, is taste masked to obscure the bitter taste of hydrocortisone and is bioequivalent to current hydrocortisone preparations. Clinical trials in young children with adrenal insufficiency demonstrated cortisol levels after dosing similar to those seen in healthy children and the drug was well tolerated and favoured over current therapy by parents. Alkindi® will provide a licenced treatment option for accurate dosing in children with adrenal insufficiency where compounded adult tablets of hydrocortisone are unsuitable.

KEYWORDS:

• Adrenal insufficiency
• congenital adrenal hyperplasia
• Paediatric Use Marketing Authorisation
• paediatrics
• granules

Box 1. Drug summary.

Declaration of interest

R.R. is a Director of Diurnal Ltd and M.W. and J.P. are employed by Diurnal Ltd. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper Supported by the European Commission under the Framework 7 Programme (Grant Agreement No. HEALTH-F5-2011-281654 -TAIN) EU FP7 Grant and Diurnal Ltd.