TIDMDNL
RNS Number : 5924V
Diurnal Group PLC
15 April 2021
15 April 2021
Diurnal Group plc
("Diurnal" or the "Company")
Request for maintenance of Orphan Designation for Efmody(R)
withdrawn
European Efmody(R) launch, planned for Q3 2021, remains
unchanged following earlier positive CHMP opinion supporting drug
approval
Granted European Efmody(R) patents provides protection until
2033
Diurnal Group plc (AIM: DNL), the specialty pharmaceutical
company targeting patient needs in chronic endocrine (hormonal)
diseases, announces that it has decided to withdraw its application
for maintenance of Orphan Designation in Europe for Efmody(R)
(hydrocortisone modified-release hard capsules - development name
Chronocort(R) ). This follows feedback from the Committee for
Orphan Medicinal Products (COMP), an advisory committee to the
European Medicines Agency (EMA). Diurnal has decided that
continuing pursuit of an Orphan Designation for the drug would be
likely to cause a significant delay in its European commercial
launch. First commercial launch is currently anticipated to be in
Q3 2021.
As previously announced by the Company on 26 March 2021, the
Committee for Medicinal Products for Human Use (CHMP), an advisory
committee of the EMA, issued a positive opinion to the European
Commission recommending Efmody(R) as treatment of adult and
adolescent patients (12 years and older) with the rare condition
congenital adrenal hyperplasia (CAH). The formal approval of
marketing authorisation from the European Commission for Efmody(R)
, which is not dependant on the maintenance of Orphan Designation,
continues to be anticipated in June 2021, in accordance with the
67-day mandated timeline following the adoption of a positive
opinion by the CHMP. In anticipation of this and commercial launch
shortly thereafter, market access activities in the Company's
target European territories are underway.
Orphan Designation provides for market exclusivity for 10 years
from launch. However, the Company currently holds granted European
patents for Efmody(R) for the treatment of patients with both CAH
and adrenal insufficiency (AI) and believes these patents provide
sufficient protection for Efmody(R) until at least 2033 in all
designated states covered by the European Patent Convention. In
addition, while Orphan Designation can be used to support premium
pricing in certain territories, Diurnal does not believe that this
is critical to the overall commercial potential of the product
given the significant clinical need for new therapies in CAH.
Efmody(R) is a preparation of hydrocortisone that has been
specifically designed for patients with CAH, an orphan condition
caused by deficiency of adrenal enzymes, most commonly
21-hydroxylase. Approximately two-thirds of CAH patients are
estimated to have poor disease control, leading to elevated
androgen levels. The condition is estimated to affect a total of
approximately 41,000 patients in Europe.
Martin Whitaker, Chief Executive Officer of Diurnal,
commented:
"Following the recent positive opinion from the CHMP we are
focussed on the timely launch of Efmody(R) in Q3 2021. Orphan
Designation for Efmody(R) in Europe is not critical to the
commercial potential of the product in this market. There remains a
strong clinical rationale for the drug's use to address the
significant need for new therapies to improve outcomes for adult
patients with CAH. This was highlighted recently in the paper in
the Journal of Clinical Endocrinology and Metabolism that detailed
our pivotal clinical trial data and results from a long term
extension study. Additionally, a robust, granted European patent
estate provides market exclusivity for the drug until at least
2033."
For further information, please visit www.diurnal.co.uk or
contact:
+44 (0)20 3727
Diurnal Group plc 1000
Martin Whitaker, Chief Executive Officer
Richard Bungay, Chief Financial Officer
Panmure Gordon (UK) Limited (Nominated Adviser +44 (0)20 7886
and Sole Broker) 2500
Corporate Finance: Freddy Crossley, Emma Earl
Corporate Broking: Rupert Dearden
+44 (0)20 3727
FTI Consulting (Media and Investor Relations) 1000
Simon Conway
Victoria Foster Mitchell
Alex Davis
Notes to Editors
About Congenital Adrenal Hyperplasia
Congenital adrenal hyperplasia (CAH) is an orphan condition
caused by deficiency of adrenal enzymes, most commonly
21-hydroxylase. This enzyme is required to produce the adrenal
steroid hormone, cortisol. The block in the cortisol production
pathway causes the over-production of male steroid hormones
(androgens), which are precursors to cortisol. The condition is
congenital (inherited at birth) and affects both sexes. The
cortisol deficiency and over-production of male sex hormones can
lead to increased mortality, infertility and issues during sexual
development including ambiguous genitalia, premature (precocious)
sexual development and short stature. Sufferers, even if treated,
remain at risk of death through an adrenal crisis.
Current therapy for CAH uses a variety of generic glucocorticoid
(steroids including hydrocortisone, dexamethasone, prednisolone and
prednisone in the US) with no standard treatment regimen.
Approximately two-thirds of CAH patients are estimated to have poor
disease control, leading to elevated androgen levels. The condition
is estimated to affect a total of approximately 41,000 patients in
Europe, with over 400,000 in the rest of the world.
About Efmody(R) (hydrocortisone modified release hard
capsules)
Efmody(R) is a preparation of hydrocortisone that has been
specifically designed to mimic the circadian rhythm of cortisol
when given in a twice-a-day "toothbrush" regimen (administered last
thing at night before sleep and first thing in the morning on
waking) to control androgen excess and chronic fatigue in patients
with diseases of cortisol deficiency. The first indication for
Efmody(R) is Congenital Adrenal Hyperplasia (CAH) in adults and
adolescents (children older than 12 years of age). Efmody(R) has
been extensively studied in 239 human subjects including 138 CAH
patients who have taken part in clinical trials in Europe and the
US.
The positive opinion from the CHMP is based on a Phase 3 study
conducted in a total of 122 patients enrolled across 11 clinical
sites, the largest ever interventional clinical trial completed in
CAH. The Phase 3 data was supported by detailed analysis of data
from an open-label safety extension study for patients completing
treatment in the Phase 3 study, which is assessing the impact of
treatment with Efmody(R) over an extended period, with a number of
patients on this trial having been treated for over 54 months.
Patient retention rates in this study have been high and patients
on this trial have, to date, shown sustained benefit from extended
Efmody(R) treatment.
About Diurnal Group plc
Founded in 2004, Diurnal is a UK-headquartered, European
specialty pharma company developing hormone therapeutics for the
global market for the life-long treatment of chronic endocrine
conditions, including congenital adrenal hyperplasia, adrenal
insufficiency and hypogonadism. 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: April 2021 Code: CORP-GB-0115
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