Generics
EffRx Pharmaceuticals Signs Exclusive License Agreement with Diurnal for the Registration and Commercialization of Efmody in Switzerland
27 April 2022 - - Switzerland-based commercial-stage company EffRx Pharmaceuticals SA has recently entered into an exclusive license agreement with UK-based Diurnal Group plc (LSE: DNL) for the registration and commercialization of Efmody as treatment for congenital adrenal hyperplasia in Switzerland, the company said.

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 our estimates, there are approximately 450 patients in Switzerland suffering from CAH.

EffRx intends to submit a Market Authorisation Application to Swissmedic as treatment for adolescent and adult patients (12 years and older) with the rare condition congenital adrenal hyperplasia 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.

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. 

Congenital adrenal hyperplasia 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.

Efmody is a preparation of hydrocortisone that has been specifically designed to mimic the circadian rhythm of cortisol when given in a twice-a-day "toothbrush" regimen (administered last thing at night before sleep and first thing in the morning on waking) to control androgen excess and chronic fatigue in patients with diseases of cortisol deficiency.

The first indication for Efmody is congenital adrenal hyperplasia in adults and adolescents (children older than 12 years of age). Efmody has been extensively studied in 239 human subjects including 138 CAH patients who have taken part in clinical trials in Europe and the US.

The MHRA and European Commission marketing authorisation approval of Efmody was based on a Phase 3 study conducted in a total of 122 patients enrolled across 11 clinical sites, including sites in Great Britain, the largest ever interventional clinical trial completed in CAH.

The Phase 3 data was supported by detailed analysis of data from an open-label safety extension study for patients completing treatment in the Phase 3 study, which is assessing the impact of treatment with Efmody over an extended period, with a number of patients on this trial having been treated for over five years.

EffRx Pharmaceuticals is a commercial-stage pharmaceutical company focused on the late stage development and commercialization of prescription medications for niche and orphan indications.

The business model is centered around providing superior clinical and commercial value propositions for physicians, payers and patients.

EffRx pro-actively seeks in-licensing opportunities for Europe in niche therapeutic areas, with a primary interest for rare diseases, where EffRx has received an orphan drug designation from the FDA.

EffRx's go-to-market competence is proven by the development, launch and lucrative expansion of Binosto in a highly competitive European market.

Our lead commercialized product, Binosto for the treatment of osteoporosis, is marketed in the US as well as selected European and Asian countries.

Diurnal Group is a European, UK-headquartered, specialty pharmaceutical company dedicated to developing hormone therapeutics to aid lifelong treatment for rare and chronic endocrine conditions, including congenital adrenal hyperplasia, adrenal insufficiency, hypogonadism and hypothyroidism.
Login
Username:

Password:


Related Headlines