MHRA authorises Diurnal’s CAH therapy Efmody

Speciality pharma company Diurnal announced today that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted Efmody a marketing authorisation in Great Britain.

Efmody (hydrocortisone modified-release hard capsules) has been cleared as a treatment for adult and adolescent patients aged 12 years and over with congenital adrenal hyperplasia (CAH).

CAH is a rare condition caused by deficiency of adrenal enzymes – usually 21-hydroxulase. This enzyme is required for the production of the adrenal steroid hormone cortisol, with the block in the cortisol production pathway causing the over-production of androgens (male steroid hormones).

In turn, the cortisol deficiency and over-productions of androgens can lead to increased mortality, infertility and issues during sexual development.

The MHRA submission for Efmody included data from a Phase III study in CAH involving 122 subjects and an open-label safety extension study.

In a statement, Diurnal said patient retention rates in the open-label study have been high and patients involved in this study have shown sustained benefit from extended Efmody treatment.

“We are pleased to have received approval for Efmody from the MHRA and look forward to making Efmody available to CAH patients in Great Britain as the first licensed treatment specifically designed to mimic the physiological circadian rhythm of cortisol,” said Martin Whitaker, chief executive officer of Diurnal.

“Today’s approval is yet another important step forward for Diurnal as we continue to drive towards becoming the world’s leading specialty endocrinology business,” he added.