EffRx Pharmaceuticals SA, a commercial-stage company that commercializes niche and orphan medicines in Switzerland and Europe, today announced it has recently entered into an exclusive license agreement with Diurnal Group plc, for the registration and commercialization of Efmody as treatment for congenital adrenal hyperplasia (CAH) in Switzerland. Under the terms of the agreement EffRx has received the exclusive rights to register and commercialize Efmody in Switzerland.
Efmody is a modified-release preparation of hydrocortisone that has been specifically designed for the treatment of patients with CAH, a rare condition caused by a genetic deficiency of adrenal enzymes. According to EffRx's estimates, there are approximately 450 patients in Switzerland suffering from CAH.
EffRx intends to submit a Market Authorisation Application (MAA) to Swissmedic as treatment for adolescent and adult patients (12 years and older) with the rare condition congenital adrenal hyperplasia (CAH) in Switzerland during the second half of 2022. The MAA submission to Swissmedic for Efmody will be based on the European regulatory dossier and published clinical trial data, with EffRx expecting potential market launch in Switzerland in 2024.
“We are excited to sign this additional agreement with Diurnal, enabling us to build on the momentum we have achieved with Alkindi in Switzerland following approval from Swissmedic in November 2021. We believe the unique release profile of Efmody, that mimics the body’s natural cortisol circadian rhythm, could have a genuine impact on CAH patients’ symptoms. We are aligned with Diurnal’s strategy to address the unmet medical need in patients suffering from diseases of cortisol deficiency and look forward to working with the Diurnal team to bring Efmody to patients suffering from CAH in Switzerland”, Lorenzo Bosisio, Chief Executive Officer of EffRx, commented.
In May 2021, Efmody was granted marketing authorisation in the European Union and was subsequently launched in Germany, Austria and the UK in September 2021. “We are pleased to deepen our relationship with EffRx to include the distribution and marketing of Efmody in Switzerland. We have been impressed by the progress EffRx has made with the regulatory approval and reimbursement of Alkindi and look forward to continuing to work with them as they prepare to submit an MAA to Swissmedic for Efmody”, Richard Bungay, Interim Chief Executive Officer of Diurnal, commented.
About congenital adrenal hyperplasia
Congenital adrenal hyperplasia (CAH) is an orphan condition caused by deficiency of adrenal enzymes, most commonly 21-hydroxylase. This enzyme is required to produce the adrenal steroid hormone, cortisol. The block in the cortisol production pathway causes the over-production of male steroid hormones (androgens), which are precursors to cortisol. The condition is congenital (inherited at birth) and affects both sexes. The cortisol deficiency and over-production of male sex hormones can lead to increased mortality, infertility and issues during sexual development including ambiguous genitalia, premature (precocious) sexual development and short stature. Sufferers, even if treated, remain at risk of death through an adrenal crisis.
Current therapy for CAH uses a variety of generic glucocorticoid (steroid) preparations including hydrocortisone, dexamethasone, prednisolone and prednisone in the US, with no standard treatment regimen. 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 16,000 patients in the US, with over 400,000 in the rest of the world.