DuoCort

Therapy

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Development Rationale

The clinical rationale for the drug development of the new therapy from DuoCort Pharma was based on the clinical need to re-establish a physiological circadian rhythm of cortisol in patients with adrenal insufficiency. The pharmaceutical development strategy was development of a novel once daily (o.d.) oral modified-release treatment regimen with an immediate release coating and an extended release core of hydrocortisone, the chemical equivalence of endogenous cortisol.

Two dose strengths of the tablet have been developed, 20 mg and 5 mg. An immediate release part provides a rapid onset of intestinal absorption and an extended release part gives a slower intestinal absorption rate and a more extended physiological plasma profile of hydrocortisone. This novel oral modified-release dosage form was developed to produce a circadian cortisol profile with a once a day oral administration during normal replacement therapy in order to produce a less variable PK profile.

Orphan Drug Designation

• An orphan drug is a medicinal product that has been developed specifically to treat a rare medical condition, i.e. an orphan disease.
• By definition, an orphan drug is one for a disease which affects less than 500 patients per million people in EU or less than 200 000 patients in the US.
• Incentives include e.g.
  • Market exclusivity – 10 years in Europe, 7 years in the USA from approval
  • Incentives for orphan drug development, such as reduced registration fees
  • Protocol assistance – approval of clinical trial design for pivotal data

DuoCort Pharma  has orphan drug designations in Europe and the US for its product for treatment of adrenal insufficiency.
For more info please see:

• DuoCort gets Orphan Drug Designation in the US for Treatment of Adrenal Insufficiency.
  
• DuoCort gets EU orphan drug designation.
  

Clinical Trial Programme

Phase I
Publication: Johannsson G, Bergthorsdottir R, Nilsson AG, Lennernäs H, Hedner T, Skrtic S. Improving glucocorticoid replacement therapy using a novel modified-release hydrocortisone tablet: a pharmacokinetic study. Eur J Endocrinol. 2009;161:119–30.

• New chronotherapy for Addison´s disease.
  
• DuoCort announces phase I data at the European Congress of Endocrinology.
  

Phase II/III
Data presented at the Endocrine Society meeting in Washington and San Diego.

• Better quality of life with new orphan drug for Addison´s disease.
  
• Succesful phase II/III trial of new therapy.
  
• www.clinicaltrials.gov
  

Phase IIIb
Trial currently ongoing. To date patients have received treatment with the new therapy for almost three years or over 150 patient years.