The European Commission (EC) has approved UK-headquartered pharma company Diurnal’s Efmody for the treatment of congenital adrenal hyperplasia (CAH).
CAH is a rare condition caused by a deficiency of adrenal enzymes, which is required for the production of the adrenal steroid hormone cortisol.
This block in cortisol production causes the over-production of male steroid hormones, meaning the condition can lead to infertility, issues during sexual development, premature sexual development, short stature and increased mortality.
Efmody is a preparation of hydrocortisone designed for patients living with CAH.
“Efmody has been proven to provide control of the disease and the overall data shows an improved hormone balance, which will provide a well-tolerated and practical twice-daily treatment regimen for these patients where there is a significant unmet need,” said Nicole Reisch, professor of internal medicine, endocrinology at the Ludwig- Maximilian-University Munich.
“As the first licensed treatment for European patients with congenital adrenal hyperplasia that mimics the physiological circadian rhythm of cortisol, we look forward to having an additional therapeutic option for congenital adrenal hyperplasia patients in the near future,” she added.
Diurnal has already started market access activities in its targeted European territories, with the first commercial launch for Efmody expected in the third quarter.
“We are delighted to have received European approval for Efmody, a significant milestone for Diurnal as our second product to receive marketing authorisation,” said Martin Whitaker, chief executive officer of Diurnal.
“We look forward to expanding our adrenal portfolio with the European launch of Efmody in Q3 2021 alongside our first product Alkindi, to provide treatment for patients with congenital adrenal hyperplasia, enabling Diurnal to continue to drive towards becoming a world-leading specialty endocrinology business,” he added.