Congenital adrenal hyperplasia treatment granted MHRA authorisation

pharmafile | July 2, 2021 | News story | Medical Communications  

The MHRA has granted marketing authorisation to Diurnal’s treatment, Efmody, as treatment of adult and adolescent patients with the rare condition congenital adrenal hyperplasia (CAH).

The approval applies in Great Britain and follows the recent authorisation from the EU.

Efmody 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 5,000 patients in Great Britain.

Professor Wiebke Arlt, Institute of Metabolism and Systems Research, University of Birmingham, said: “I am excited to see Efmody approved in Great Britain as a treatment for patients with CAH, a rare condition where patients and doctors need to face the challenge of deficient cortisol production and adrenal androgen excess.

“Efmody has been developed for these patients and I believe this drug approval provides a new option for the treatment of CAH patients, where there is a significant unmet medical need.”

Martin Whitaker, Chief Executive Officer of Diurnal, said: “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.

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.”

Following feedback from the MHRA and consistent with the approach taken in the EEA, Diurnal has decided not to pursue Orphan Drug Status for Efmody in Great Britain as it would likely cause a significant delay to the commercial launch.

Lilly Subbotin

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