NEWS FLOW

20th December 2016
Diurnal announces submission of European Paediatric Use Marketing Suthorisation application for Infacort®
(Hydrocortisone Granules)
TAIN consortium member Diurnal announced the submission of a Paediatric Use Marketing Authorisation (PUMA) application for Infacort® to the European Medicines Agency (EMA). This application has completed the validation period and has been accepted for review by the EMA.


This submission follows the highly statistically significant data previously reported from the Company's pivotal Phase III study for Infacort® in Europe for paediatric Adrenal Insufficiency (AI). The EMA has already approved a Paediatric Investigation Plan (PIP) for Infacort®, setting out the regulatory pathway to market authorisation via the PUMA route. If approved, Infacort® has the potential to be the first licensed treatment in Europe for AI (including the related condition Congenital Adrenal Hyperplasia (CAH))

 
 

12th October 2016
Diurnal announces full results from positive European Infacort®
Phase III study
The TAIN consortium, was pleased to announce full results from the Phase III European study for Infacort® in paediatric Adrenal Insufficiency (AI). Positive headline data from this pivotal study were published on 11 July 2016 and today's update follows a comprehensive analysis of the data.   


The Phase III trial was designed in agreement with the European Medicines Agency (EMA) and conducted in a total of 24 neonates and children under the age of six years who require replacement therapy for AI due to Congenital Adrenal Hyperplasia (CAH), primary adrenal failure or hypopituitarism. 

Comprehensive analysis of the results confirms that the study met its primary endpoint, demonstrating a statistically significant (p<0.0001) increase in cortisol values following administration of Infacort® compared to pre-dose values. The median 60‑minute cortisol level achieved after Infacort® treatment was 535 nmol/L, which is comparable to the mean peak morning cortisol of healthy children.

 
 

11th July 2016
Diurnal reports positive headline data from European Infacort® Phase III pivotal study

TAIN consortium member Diurnal announced, ahead of schedule, positive headline data from the pivotal study for Infacort® in Europe for paediatric Adrenal Insufficiency (AI).


The Phase III trial was designed in agreement with the European Medicines Agency (EMA) and conducted in a total of 24 subjects before their sixth birthday, requiring replacement therapy for AI due to Congenital Adrenal Hyperplasia (CAH), primary adrenal failure or hypopituitarism.

Initial analysis of the results confirms that the study met its primary endpoint, demonstrating a statistically significant (p<0.0001) increase in cortisol values following administration of Infacort® compared to the pre-dose values. No serious adverse events were reported. A full evaluation of the data is underway and the results of this comprehensive analysis are expected later this quarter.

 
 

19th May 2015
FDA grants orphan drug designation to Diurnal for Infacort®

TAIN partner Diurnal announced that it has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) for Infacort®, an oral hydrocortisone formulation for the treatment of paediatric Adrenal Insufficiency ("AI"). The designation provides seven years of market exclusivity in the US after market authorisation and follows the recent Orphan Drug Designation granted by the FDA for Chronocort®, Diurnal's complementary product for adult Congenital Adrenal Hyperplasia, a related condition.

 

30th March 2015

The TAIN Consortium enrols first patient onto Paediatric Phase 3 Study in Europe

Diurnal, a member of the TAIN Consortium - a collaborative European research programme - is pleased to announce today that the first patient has been enrolled onto a Phase 3 trial of its paediatric product - Infacort® (immediate release hydrocortisone).  The Infacort 003 trial is a European registration trial in paediatric patients who are less than six years of age with the rare condition Adrenal Insufficiency. The Infacort® trial is scheduled to be completed by Q3, 2016.

About Adrenal Insufficiency
Adrenal Insufficiency (AI) is a condition characterised by deficiency in cortisol, an essential hormone in regulating metabolism and the response to stress. AI has been identified as an orphan disease in Europe where there are estimated to be approximately 4,000 sufferers younger than the age of six. Currently there are no licensed hydrocortisone preparations in Europe to treat these young patients with children often administered compounded adult tablets or other unlicensed products.  Poor control of disease can result in precocious puberty in young children leading to virilisation and infertility, combined with fatigue and a poor quality of life in adulthood.

About Infacort®
Infacort® represents the first-ever preparation of hydrocortisone specifically designed for use in children suffering from AI. It is a patented, immediate-release, oral, paediatric formulation of hydrocortisone that allows for release of cortisol in a manner that mimics current clinical practice. This therapeutic approach has the potential to help young patients less than six year of age suffering from diseases due to cortisol deficiency including adrenal insufficiency. AI requires life-long treatment and this novel approach to drug delivery has the potential to significantly improve these young patients' lives. The European Medicines Agency has already approved a Paediatric Investigation Plan (EMEA-001283-PIP01-12) for Infacort®, which sets out the regulatory pathway to market authorisation via the Paediatric Use Marketing Authorisation (PUMA) route.

Martin Whitaker, Scientific Coordinator,said:
"The start of the Infacort® study marks a significant achievement by the TAIN Consortium. We are delighted to have reached this development milestone and are very excited about the potential opportunity for this product. This Phase 3 trial has the potential to demonstrate the safety and efficacy profile of a new important therapy that could deliver patient benefits to young children living with adrenal insufficiency in Europe".

Please direct all enquiries to: martin.whitaker@sheffield.ac.uk



20th June 2013
Successful completion of Infacort®001 study
The TAIN Consortium today announced positive results from its Infacort 001 clinical study. The study in 16 healthy adult volunteers, forms part of the company's Infacort® development programme to develop a paediatric treatment for children under 6 years of age suffering from the rare disease Adrenal Insufficiency.

The trial successfully met its primary objective of demonstrating Infacort® bioequivalence to 10mg hydrocortisone tablets in healthy adult volunteers. Infacort® was shown to be safe, well-tolerated and to be of neutral taste.

Infacort® is a paediatric hydrocortisone product being developed under the European Commission funded collaborative research Framework 7 Programme ' "Treatment of Adrenal Insufficiency in Neonates (TAIN)". The programme will, for the first time, create a regulatory-approved paediatric version of hydrocortisone.

About Adrenal Insufficiency in Children
Adrenal Insufficiency is a rare disorder that prevents the body from producing sufficient amounts of the essential hormone cortisol. Hydrocortisone is a glucocorticoid hormone used as a replacement therapy for the treatment of Adrenal Insufficiency. The problem of effective hydrocortisone replacement is particularly acute among young patients (neonates and infants) for whom no licensed therapy exists. Current therapy requires adaption of adult formulations through, amongst other methods, crushing tablets. This is problematic and gives variable and inaccurate dosing as crushed tablets are relatively insoluble, provide an unpredictable pharmacokinetic profile, have no established stability, and are inconvenient for clinicians and parents. Current hydrocortisone preparations put the neonate at risk over and under treatment with potential long term health consequences including obesity, ill health and sudden death.

About Infacort®
Infacort® is an immediate release hydrocortisone granule preparation specifically designed for use in children under 6 years of age. Infacort® is being developed under a Paediatric Investigation Plan approved by the European Medicines Agency (EMA) EMEA-001283-PIP01-12. Infacort® has been designed with taste masking excipients that are acceptable for paedriatric use and in a variety of dosage units to give maximum flexibility to clinicians to tailor the treatment to the child.

About TAIN
The TAIN Consortium involves European leaders in drug development, neonatology and paediatric pharmacology. The approved Paediatric Investigation Plan from the EMA will enable clinical trials to be carried out to provide sufficient evidence of safety and efficacy for Infacort® to allow submission of a Paediatric Use Medicines Authorisation (PUMA). The project also aims to raise awareness of Adrenal Insufficiency to maximise its positive impact for patients, parents and clinicians. For further information see www.tain-project.org.

Martin Whitaker, Scientific Coordinator said:
"These results for Infacort® were excellent and hit all the set target success parameters. We hope to progress to Phase 3 studies rapidly. This is a very exciting time for the whole of the TAIN Consortium team."
 
Please direct all enquiries to: martin.whitaker@sheffield.ac.uk



20th June 2013
Paediatric Investigation Plan for Infacort approved by the European Medicines Agency. 

A Paediatric Investigation Plan has been approved from the European Medicines Agency (EMA) for a new formulation of hydrocortisone. Infacort® will be developed for the treatment of children under 6 years old suffering the inability to produce cortisol.

The problem of effective hydrocortisone replacement is especially acute in this paediatric setting where no licensed therapies exist.  Under the Paediatric Investigation Plan, clinical trials will be carried out to provide sufficient evidence of safety and efficacy for Infacort to allow submission of a Paediatric Use Medicines Authorisation (PUMA) to the European Medicines Agency by the end of 2015 and its subsequent use in patients.

Infacort® is being developed by partners under the EU collaborative research programme - "Treatment of Adrenal Insufficiency in Neonates (TAIN)". The programme will, for the first time, create a regulatory-approved paediatric version of hydrocortisone.

Participants
Diurnal Limited, United Kingdom
SimCyp Limited, United Kingdom
The University of Sheffield, United Kingdom
Glatt GmbH, Germany
ADD Advanced Drug Delivery Technologies, Switzerland
Charité, Universitätsmedizin Berlin, Germany