University spin-out granted first licensed treatment to help children with rare disorder
- First licensed treatment in Europe specially designed for use in children with adrenal insufficiency
- Adrenal insufficiency means not enough cortisol is produced – a potentially fatal deficiency
- University spin-out company’s first product receives marketing authorisation from the European Commission
A University of Sheffield spin-out company has been granted its first licensed treatment in Europe for children with a rare disorder.
Diurnal Group plc, which develops innovative therapies for patients with chronic hormonal diseases, was formed based on research by Professor Richard Ross, an expert in Endocrinology from the University of Sheffield’s Department of Oncology and Human Metabolism.
The European Commission granted Diurnal a paediatric use marketing authorisation (PUMA) for Alkindi® - as replacement therapy for adrenal insufficiency in infants, children and adolescents - following the positive opinion issued by the European Medicines Agency.
Paediatric Adrenal Insufficiency (Paediatric AI) is a rare disorder which affects approximately 4,000 children under the age of six in Europe.
Adrenal Insufficiency is where the adrenal glands, which sit on the top of the kidneys, are damaged and not enough cortisol and aldosterone are produced. Cortisol is an essential stress hormone and a deficiency in cortisol can be fatal unless the hormone is replaced by the pharmaceutical form, hydrocortisone.
Children with adrenal insufficiency must take hydrocortisone replacement therapy from birth, however to date there has been no satisfactory paediatric formulation of hydrocortisone and parents have had to crush adult tablets. This poses a risk of over or under dosing of children which could lead to adrenal crises and long-term poor health outcomes.
Research developed by the University of Sheffield identified the problem and with the spin-out company Diurnal has developed a paediatric formation of hydrocortisone, Alkindi®, specially designed so it can be taken from birth.
Professor Richard Ross, Professor of Endocrinology at the University of Sheffield and Chief Scientific Officer of Diurnal, said: “I am delighted that this medicine, specifically designed to treat neonates, infants and children with adrenal insufficiency, may soon be available to patients in Europe, following market authorisation approval by the European Commission.
“This represents the culmination of six years intense research and development to address a major unmet need in children born with adrenal insufficiency.
“Alkindi® provides a safe, easy to administer, paediatric designed formulation which I believe will reduce the risk of adrenal crises and improve the treatment and long-term outcomes of children with adrenal insufficiency.”
The research was awarded a €4.2 million grant from the European Commission in collaboration with Diurnal to produce the formulation which is based on multi-particulates (granules) - beads about 0.5mm in diameter. Each bead is coated with hydrocortisone and then a taste masking layer that obscurers the bitter taste of hydrocortisone. Using a multi-particulate ensures doctors can provide the small doses required for neonates that can then be increased as the child grows.
Clinical trials in neonates, infants and children aged up to six years have shown Alkindi® is safe, effective and well tolerated with children, with parents preferring Alkindi® to their current treatment.
Professor Martin Whitaker, Honorary Professor of Medical Innovation at the University of Sheffield and Chief Executive Officer of Diurnal, said: “We are delighted to receive European approval for Alkindi ®, a significant milestone for Diurnal as our first marketing authorisation.
“This approval is a major breakthrough for paediatric patients with adrenal insufficiency, with Alkindi ® being the first licensed treatment in Europe specifically designed for use in these children.
“Paediatric AI is currently treated by the unlicensed compounding adult preparations of hydrocortisone. We have a detailed commercial plan in place to bring this important new treatment to paediatric patients and address the current unmet medical need. We anticipate the first launch of Alkindi ® in 2018.
“Alkindi® joins the exclusive list of one of the few medicines to be successfully approved and commercialised by a UK company based on research from a UK University. ”
Key facts: Adrenal insufficiency
- Adrenal insufficiency results from failure of the adrenal gland to secrete the stress hormone cortisol and without cortisol replacement adrenal insufficiency is fatal with patients dying in an adrenal crisis.
- The commonest cause of adrenal insufficiency in young children is congenital adrenal hyperplasia where a baby is born with a mutation in the gene responsible for the production of cortisol.
- These babies need treatment from birth. Adrenal insufficiency can be acquired at any age either due to failure of the adrenal or failure of the pituitary that stimulates the adrenal.
- About 8,400 people in the UK have adrenal insufficiency.
The University of Sheffield
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