FREIENBACH, Switzerland–(BUSINESS WIRE)–#Alkindi–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 our 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.
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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.
About Efmody® (hydrocortisone modified-release hard capsules)
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 (CAH) 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. Summary of Product Characteristics (SmPC) for UK (Northern Ireland) can be found here.
About EffRx Pharmaceuticals
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 (ODD) 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.
About Diurnal Group plc
Diurnal Group plc 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. Its expertise and innovative research activities focus on circadian-based endocrinology to yield novel product candidates in the rare and chronic endocrine disease arena.
For further information about Diurnal, please visit www.diurnal.com
Contacts
Media:
Ms. Pamela Saredi
psaredi@effrx.com