DuoCort Gets Orphan Drug Designation in the US for Treatment of Adrenal Insufficiency
- Details
- Published on Friday, 27 June 2008 01:00
Helsingborg, Sweden, June 27, 2008 – DuoCort Pharma AB, a privately held Swedish drug development company, today announced that the US Food and Drug Administration (FDA) has granted an Orphan Drug Designation for the company’s DuoCort™ hydrocortisone dual-release oral tablet in development for the treatment of adrenal insufficiency – the inability of the body to produce sufficient amounts of the essential hormone cortisol.
Orphan drug designation will give DuoCort™ market exclusivity in the US
for seven years after the product attains marketing authorization.
"Current research confirms that conventional therapy is suboptimal and
is not serving patients as well as it should. There is scope for
improvement in the delivery of cortisol replacement and we are
convinced DuoCort™ will offer a better treatment option for patients
with adrenal insufficiency and deliver better long-term outcomes,” said
Dr. Gudmundur Johannsson, Chief Medical Officer of DuoCort
Pharma.“Orphan designation in the US will facilitate this development
aimed at demonstrating the significant benefit of cortisol replacement
which effectively mimics the physiological diurnal release profile of
cortisol in a convenient once-daily tablet. The new DuoCort™ therapy
will be the first real innovation for adrenal insufficiency patients in
over 30 years,” continued Dr. Johannsson.The orphan-designated product
is a novel, dual-release, oral hydrocortisone tablet in 5 mg and 20 mg
dosage strengths currently in late stage clinical trials in Europe.
DuoCort™ will improve today’s cortisol replacement therapy with a
once-daily tablet that delivers a more physiological dose of the active
substance, better mimicking the body’s own cortisol serum
profile.DuoCort™ 5 mg and 20 mg dual-release hydrocortisone tablets are
currently undergoing a pivotal clinical Phase II/III trial in Europe
assessing the pharmacokinetics, safety and tolerability of once-daily
DuoCort™ in comparison to conventional thrice-daily oral hydrocortisone
therapy in patients with adrenal insufficiency. The trial commenced in
August 2007 and is being conducted at five University endocrinology
clinics. Data from the study is expected to be available in the second
half of 2008. With normal regulatory review and approval times,
DuoCort™ could be on the market by 2010.
Orphan Drug legislation in
the US provides incentives to encourage the development and marketing
of medicines for rare diseases. Designation as an orphan medicinal
product does not indicate that the product has yet fulfilled the
efficacy, safety and quality criteria required for drug marketing.
These remain to be fulfilled in the pharmaceutical and clinical
development of the drug and assessed by the FDA at the marketing
authorisation stage.
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