Category Archives: Uncategorized

EffRx Attending the Annual Meeting SSEDP / SGPED on 26. January 2023 at the Universitäts-Kinderspital Zürich

The Swiss Society for Pediatric Endocrinology and Diabetology (SGPED) is composed of pediatric endocrinologists and individuals from allied health professions. Together with supporting sponsors like EffRx, the Society is jointly committed to improving the care of children and adolescents with endocrinological diseases and diabetes mellitus. For the second year in a row, EffRx will be present at the annual meeting of SGPED, taking place this year in Zürich.

EffRx Attends the 1st Eastern Switzerland Symposium on Rare Diseases in St. Gallen, Switzerland

The Eastern Switzerland Symposium on Rare Diseases took place for the first time on November 24, 2022, and attracted a high number of participants from a broad range of disciplines. The motto of the conference, chaired by experts from the Cantonal Hospital St. Gallen and Eastern Switzerland Children’s Hospital, was “Learning from the Rare Diseases for the Common Ones”. The symposium offered the opportunity to learn more about rare diseases from competent speakers and engage in productive discussions. Having the patient perspective in mind, exchanges focused on recent and future development of diagnostics and patient care in patients with rare diseases.

EffRx Pharmaceuticals announces the launch of Alkindi® as replacement therapy for pediatric adrenal insufficiency in Switzerland

FREIENBACH, Switzerland – EffRx Pharmaceuticals SA, a commercial-stage company that commercializes niche and orphan medicines in Switzerland and Europe, today announces the launch of Alkindi® (hydrocortisone capsules for oral use) in Switzerland as replacement therapy for adrenal insufficiency in infants, children and adolescents (from birth to <18 years old).

Alkindi® is the first approved pediatric-specific hydrocortisone formulation for the replacement therapy for adrenal insufficiency (AI) in pediatric patients. Alkindi® is available in color-coded capsules with four different dose strengths (0.5 mg, 1 mg, 2 mg, 5 mg) and allows for individualized, accurate and flexible dosing – without the need for magisterial formulations or tablet fragmentation.

Pediatric AI, including the related genetic condition congenital adrenal hyperplasia (CAH), is a condition characterized by a deficiency in cortisol. Untreated, the disease is associated with significant morbidity and increased mortality. In Switzerland, there are approximately 200 patients suffering from pediatric AI. This condition in children is currently treated by compounding hydrocortisone or crushing/splitting tablets of adult preparations with the potential risks of lack of efficacy and/or increased side effects.

Dr. Lorraine Zakin, Senior Director Medical Affairs at EffRx commented: “I am very pleased that this medicine, specifically studied in children and designed to treat children with adrenal insufficiency, is available in Switzerland. Alkindi® addresses the shortfall in pediatric-specific medicines.”

Alkindi® advances the therapeutic options to help young patients suffering from pediatric AI and CAH and provides the first regulated, consistent preparation of hydrocortisone to ensure efficacy, safety and ease of use for children suffering from AI and their caregivers.

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About Pediatric Adrenal Insufficiency (AI) and congenital adrenal hyperplasia (CAH)

Pediatric AI and the related genetic condition CAH are characterized by deficiency in cortisol, an essential hormone in regulating metabolism and the response to stress. The primary symptoms of AI are chronic fatigue, risk of adrenal crisis and death in case of inadequate cortisol replacement.

About Alkindi®

Alkindi® is the first preparation of hydrocortisone specifically designed for use in children suffering from pediatric AI. Alkindi® is a patented, oral, immediate-release pediatric formulation of hydrocortisone granules in capsules for opening that allows for accurate, yet flexible and personalized dosing in children. This therapeutic approach has the potential to help young patients less than eighteen years of age suffering from pediatric AI and the related condition CAH.

About EffRx Pharmaceuticals

EffRx Pharmaceuticals is a commercial-stage pharmaceutical company focused on the late-stage development and commercialization of medicines for niche and orphan indications. The business model is centered around providing superior clinical value propositions for physicians, payers and patients.

EffRx pro-actively seeks in-licensing opportunities for Switzerland and Europe in niche therapeutic areas, with a primary interest for rare diseases. EffRx’s go-to-market competence is proven by the development, launch and expansion of Binosto® in highly competitive European markets as well as US, selected MEA and Asian countries.

EffRx Attending the 9th I-DSD Symposium 2022 in Bern, Switzerland, University Hospital Bern from 14-16th July 2022

The International Symposium on the Differences & Disorders of Sex Development I-DSD has been very successful in attracting a rapidly expanding multidisciplinary group of experts, eager to discuss the latest advances in the field and have laid the foundation for fruitful international collaborations.

In 2021, the I-DSD/I-CAH/I-TS has a network that reaches 260 centers in 63 countries on all continents. Of these, 115 centers from 40 countries use the registries and have entered over 5,500 cases where information can be shared for a range of purposes that have the ultimate aim of improving the health of people with these rare conditions.

World Orphan Drug Alliance – combining forces to reach patients with rare diseases across the globe

  • World Orphan Drug Alliance (WODA) is a new global alliance of full-service regional distributors for orphan drugs.
  • WODA covers 68 countries on 4 continents.
  • There are more than 64 million potential patients with rare diseases in the WODA regions.

Zurich, Ljubljana, Moscow, Sao Paulo, Dubai, May 12, 2022 EffRx Pharmaceuticals, Medis, Orpharm, OrphanDC, and Vector Pharma, announced today the newly formed World Orphan Drug Alliance (WODA). WODA aims to provide comprehensive support to pharmaceutical and biotech companies with rare diseases, oncology and highly specialized therapeutics portfolio, starting from named patient programs through to full commercialization.

By bringing together experts for rare diseases, WODA has capabilities to provide all the services necessary for orphan medicines to reach patients in need, replacing local branches of global distributors or multinational companies.

Under WODA alliance we are offering future partners the opportunity to cover our entire territory or cherry-pick the territories they require, while using local teams in each territory with the local knowledge and skillset in the therapeutic area we cover,” commented Vector Pharma Managing Director Samer Semaan. “Due to our flexible setup, we have the ability to adjust to partners’ needs instantaneously and provide a tailored solution.”

In total, WODA covers 68 countries in the following regions: Turkey, Middle East and North Africa, Russia and CIS region, Central and Eastern Europe, Latin America and Switzerland.

WODA focuses on the markets, that are complex and often overlooked by big pharmaceutical and biotech companies. However, WODA territories provide both, a pool of patients in need and potential funding opportunities for rare and specialized diseases. There are potentially more than 64 million rare disease patients in the WODA regions,” adds Martina Perharič, Medis CEO.

About Founding Partners:

  • EffRx Pharmaceuticals is a Switzerland based company focused on the late-stage development and commercialization of prescription medications for niche and orphan indications.
  • Medis based in Slovenia is the commercialization partner of choice for innovative pharmaceutical and biotech companies seeking strong business growth in Central and Eastern Europe.
  • Orpharm is a Moscow based full-service distributor covering Russia and the Commonwealth of Independent States (CIS).
  • OrphanDC based in Sao Paolo, Brazil acts as a partner for biotech companies in Latin America. They focus on supporting their clients from the clinical development stage throughout the product lifecycle.
  • Vector Pharma is a Dubai based full-service distributor covering Middle East, North Africa and Turkey.

More about WODA: www.woda-alliance.com

 

EffRx Pharmaceuticals Signs Exclusive License Agreement with Diurnal for the Registration and Commercialization of Efmody® in Switzerland

FREIENBACH, Switzerland –-BUSINESS WIRE–-  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 a 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®, which 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.

Media contact: Ms. Pamela Saredi, psaredi@effrx.com

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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

EffRx Supports the 15th Annual Rare Disease Day on 28. February 2022

Rare Disease Day is the globally-coordinated movement on rare diseases, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.

Since its creation in 2008, Rare Disease Day has played a critical part in building an international rare disease community that is multi-disease, global, and diverse– but united in purpose.

Rare Disease Day is observed every year on 28 February (or 29 in leap years)—the rarest day of the year.
Rare Disease Day was set up and is coordinated by EURORDIS and 65+ national alliance patient organisation partners. Rare Disease Day provides an energy and focal point that enables rare diseases advocacy work to progress on the local, national and international levels.

To learn more visit rarediseaseday.org

EffRx Obtains Swiss Marketing Authorization for Alkindi® for Paediatric Adrenocortical Insufficiency

FREIENBACH, Switzerland –-BUSINESSWIRE— EffRx Pharmaceuticals SA, a commercial-stage company that commercializes niche and orphan medicines in Switzerland and Europe, today announced that Swissmedic has approved Alkindi® for the treatment of paediatric adrenocortical insufficiency (AI).

Alkindi®, developed by Diurnal Group plc, is the first preparation of hydrocortisone specifically designed for use in children suffering from paediatric adrenocortical insufficiency (AI). Alkindi® is a patented, oral, immediate-release paediatric formulation of hydrocortisone granules in capsules for opening that allows for accurate age-appropriate dosing in children. This therapeutic approach has the potential to help young patients less than eighteen years of age suffering from paediatric AI and the related condition congenital adrenal hyperplasia (CAH).

Alkindi® is approved and marketed in the European Union and is the first preparation of hydrocortisone specifically designed for use in children suffering from AI. On September 29th, 2020 the US Food and Drug Administration (FDA) has also approved Alkindi® for AI. This new treatment approach is expected to be available on the Swiss market by H1 2022. In Switzerland there are approximately 200 patients suffering from paediatric AI.

The marketing authorisation approval in Switzerland is based upon a pivotal open-label Phase III clinical trial conducted in 24 children, requiring replacement therapy for adrenal insufficiency due to congenital adrenal hyperplasia or hypopituitarism. The study successfully met its primary endpoint and no serious adverse events were reported.

“We are delighted to have received Swissmedic approval for Alkindi®. This approval is a major breakthrough for paediatric patients with adrenal insufficiency, with Alkindi® being the first licensed treatment in Switzerland specifically designed for use in infants, children and adolescents (from birth to less than 18 years old)” commented Lorraine Zakin, Senior Director Medical Affairs at EffRx Pharmaceuticals.

Paediatric AI and the related genetic condition CAH is a condition characterised by deficiency in cortisol, an essential hormone in regulating metabolism and the response to stress. The primary symptoms of AI are chronic fatigue and patients are at risk of adrenal crisis and death if they do not have adequate cortisol replacement. AI is either primary or secondary, with primary AI resulting from diseases intrinsic to the adrenal gland and secondary AI resulting from pituitary diseases where there is a failure of stimulation of the adrenal by the pituitary of the signalling hormone ACTH (adrenocorticotropic hormone).

About EffRx Pharmaceuticals SA

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 one of its pipeline assets has received an orphan drug designation (ODD) from the FDA in US.

About Diurnal Group plc

Founded in 2004, Diurnal is a UK-headquartered, European specialty pharma company developing high quality products for the global market for the life-long treatment of chronic endocrine conditions, including congenital adrenal hyperplasia, adrenal insufficiency and hypogonadism. Its expertise and innovative research activities focus on circadian-based endocrinology to yield novel product candidates in the rare and chronic endocrine disease arena.

Media contact: Ms. Pamela Saredi, psaredi@effrx.com