Press Releases
19.09.2023
EffRx Pharmaceuticals Signs Exclusive Licensing Agreement With Radius Health To Commercialize BINOSTO® In The United States
Freienbach, September 19th, 2023 – EffRx Pharmaceuticals SA, a commercial-stage pharmaceutical company focused on the development and commercialization of musculoskeletal and rare diseases medications, today announced that it has signed an exclusive licensing and distribution agreement with Radius Health, Inc for Binosto® for the United States.
BINOSTO® is indicated for the treatment of osteoporosis in postmenopausal women and to increase bone mass in men with osteoporosis. BINOSTO® provides an important once-weekly treatment option, particularly for patients with osteoporosis who may not be able to take alendronate tablets. Radius is a major player in the U.S. bone market and currently markets TYMLOS® for the treatment of osteoporosis in the U.S. BINOSTO® expands Radius’ presence in the treatment of bone health with a second product for the approximately 10 million people in the U.S. living with osteoporosis.
Scott Briggs, Chief Executive Officer of Radius, commented, “We are pleased to offer another product to support the needs of patients who are living with osteoporosis. We have seen strong growth of TYMLOS® in the last year, and while it will remain a primary focus, BINOSTO® addresses an important unmet need particularly for patients who have difficulty swallowing pills.”
Ed Cinca, Chief Commercial Officer added, “The addition of BINOSTO® along with our currently marketed product TYMLOS® will allow Radius to expand its product offerings to healthcare providers treating osteoporosis. Given our success with TYMLOS®, we believe that we are well-positioned to support the future growth of BINOSTO®, which has been particularly successful in markets outside the U.S. We look forward to the introduction of BINOSTO in the coming months.”
Lorenzo Bosisio, Chief Executive Officer of EffRx, said, “We are thrilled to announce the partnership with Radius. With their well-established expertise and footprint in the U.S. osteoporosis market, Radius is the partner of choice to make Binosto® widely available to U.S. patients suffering from this debilitating bone-thinning disease.”
Under the terms of the agreement, EffRx will grant Radius an exclusive, royalty bearing license to BINOSTO® in the U.S. and will also supply product to Radius.
Destum Partners, Inc. acted as exclusive advisor to EffRx for the transaction.
About BINOSTO®:
BINOSTO® (alendronate sodium) is an effervescent tablet for oral solution indicated for the treatment of osteoporosis in postmenopausal women and treatment to increase bone mass in men with osteoporosis. BINOSTO® has been shown to increase bone mass and reduce the incidence of fractures. Each dissolving tablet contains the same strength ingredient as once weekly Fosomax®. BINOSTO® is administered once weekly. The most common adverse events associated with BINOSTO® include abdominal pain, acid regurgitation, constipation, diarrhea, dyspepsia, musculoskeletal pain, and nausea. Please see the full prescribing information for details.
About EffRx:
EffRx is a Swiss commercial-stage pharmaceutical company focused on the development and commercialization of musculoskeletal and rare diseases medications. EffRx’s lead commercialized product, Binosto® for the treatment of osteoporosis, is successfully commercialized by Licensees across European, Asian and Middle Eastern countries. EffRx is controlled by Abiogen Pharma S.p.A., an Italian leading company in the field of osteoarticular and bone metabolism diseases.
About Radius:
Radius is a global biopharmaceutical company focused on addressing unmet medical needs in bone health and related areas. Radius’ lead product, TYMLOS® (abaloparatide) injection, was approved by the U.S. Food and Drug Administration in April 2017 for the treatment of postmenopausal women with osteoporosis at high risk for fracture, and in December 2022 for the increase in bone density in men with osteoporosis at high risk for fracture.
Contact:
Media: Ms. Pamela Saredi
info@effrx.com
29.05.2023
Abiogen Pharma Completes the Acquisition of EffRx
EffRx Pharmaceuticals SA joins forces with Abiogen Pharma S.p.A.
Read PR: Abiogen & EffRx Press Release
20.07.2022
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 a 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 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.
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.
20.07.2022
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 skill set 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
26.04.2022
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 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.
Media contact: Ms. Pamela Saredi, psaredi@effrx.com
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 in 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
16.11.2021
EffRx Obtains Swiss Marketing Authorization for Alkindi® for Paediatric Adrenocortical Insufficiency
FREIENBACH, Switzerland –-BUSINESS WIRE— 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
04.11.2021
EffRx Obtains Swiss Marketing Authorization for Bronchitol® in Cystic Fibrosis
FREIENBACH, Switzerland–(BUSINESS WIRE)–EffRx Pharmaceuticals SA, a commercial-stage company that commercializes niche and orphan medicines in Switzerland and Europe, today announced that Swissmedic has approved Bronchitol® (inhaled mannitol) for the treatment of cystic fibrosis (CF) in adults and in children aged 6 years and above as add‐on to other medicines.
Bronchitol® is a precision spray‐dried form of mannitol that is delivered to the lungs by a specially designed, portable inhaler. Bronchitol® improves lung function and helps to clear mucus from the lungs in patients suffering from cystic fibrosis. Patients are required to pass a tolerance test prior to being prescribed Bronchitol® to ensure that they are not hyperresponsive to mannitol.
Bronchitol® is currently marketed in Europe, Russia, Australia and the United States and was developed by Pharmaxis Ltd, a listed pharmaceutical research company in Australia. The clinical program supporting the registration of Bronchitol® consisted of three large-scale global clinical trials and enrolled a total of 1,065 subjects. Bronchitol® use led to a sustained improvement in FEV1 (Forced Expiratory Volume) versus control. The statistically significant improvement in FEV1 was observed over the 26-week treatment period in those patients receiving Bronchitol® when compared to patients in the control group. The most common (≥1/100, <1/10) adverse reactions include cough, hemoptysis, oropharyngeal pain, vomiting, wheezing and headache.
“We’re excited to have achieved this important milestone and that patients living with cystic fibrosis will be able to benefit from this treatment in Switzerland. Bronchitol® offers a portable and discreet option for CF management, with no routine cleaning or maintenance of the inhaler device required,” said Lorraine Zakin, Senior Director Medical Affairs at EffRx Pharmaceuticals.
Cystic fibrosis (CF) is an inherited, life-limiting disease that affects the body’s exocrine glands, which produce mucus, saliva, sweat and tears. In the lungs of a CF patient, the thick mucus and the thinning of the airway surface liquid make it nearly impossible for the cilia to clear bacteria from the airway. According to the Swiss Society for Cystic Fibrosis (CFCH), around 320,000 Swiss people are carriers of an altered gene that can cause CF (approximately one in 25 people)[1].
EffRx expects the availability of Bronchitol® in Switzerland in the second half of 2022.
[1] Was ist Cystische Fibrose | Cystische Fibrose Schweiz
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 Switzerland and Europe in niche therapeutic areas, with a primary interest for rare diseases, where EffRx had received an orphan drug designation (ODD) from the FDA.
About Pharmaxis
Pharmaxis Ltd is an Australian pharmaceutical research company developing drugs for inflammatory and fibrotic diseases, with a focus on myelofibrosis. The company has a highly productive drug discovery engine built on its expertise in the chemistry of amine oxidase inhibitors, with drug candidates in clinical trials. Pharmaxis has also developed two respiratory products which are approved and supplied in global markets, generating ongoing revenue. Pharmaxis is listed on the Australian Securities Exchange (PXS). Its head office, manufacturing and research facilities are in Sydney, Australia.
22.06.2021
A Prospective Study in More Than 1,000 Osteoporosis Patients Concluded That Binosto® (Buffered Soluble Alendronate) May Increase Patient Satisfaction, Long-Term Adherence and Therefore Efficacy
FREIENBACH, Switzerland–(BUSINESS WIRE)–
Corporate IR Press Release
In a newly published international multicenter study1 in over 1,000 osteoporosis patients treated with Binosto® and followed for 12 ± 3 months,
- Binosto was well tolerated
- Treatment persistence was high
- Mean overall compliance was high too
- Patient satisfaction remained at approximately 90%
EffRx Pharmaceuticals SA, a commercial-stage pharmaceutical company that commercializes niche and orphan medicines in Switzerland and Europe, announces the full paper publication of the GastroPASS Study Results of Binosto® (buffered soluble alendronate) in JBMR® Plus, a premier peer-reviewed medical journal. Prof. Salvatore Minisola will present the study results as an oral communication (OC 19) at the WCO-IOF-ESCEO2 virtual congress on August 28, 2021 at 11:40 am.
GastroPASS was a prospective, observational, multicenter, multinational, single-arm, post-authorization safety study. The study enrolled 1,084 postmenopausal women with osteoporosis, newly prescribed Binosto® and naïve to bisphosphonate therapy. At baseline, 31% of enrolled patients reported a medical history of gastrointestinal (GI) tract conditions that did not exclude study participation. Patients were followed for 12 ± 3 months. The primary objective was to evaluate the incidence of upper GI adverse events and medication errors during treatment. Secondary aims were to evaluate persistence, discontinuation and reasons for discontinuation.
The cumulative incidence of all upper GI AEs related to Binosto® observed during the duration of the study was low at 9.6%. The study authors stated that “the incidence of upper GI AEs in this “real-world” study were found to be lower than that observed in alendronate-treated groups from randomized controlled trials with a duration of 12 months.” Also, the incidence rate of individual gastric AEs related to Binosto was low (ranging from 0 to 2.1 per 1,000 patients/month) and decreased over the three follow-up periods of the study. No events for gastric ulcer, gastric perforation, gastric hemorrhage, and gastric stenosis were observed.
Treatment persistence reported in this study was high at 79.7% at 12 ± 3 months. These results are well in line with the recently published findings by Giusti et al.3 Mean overall compliance was high, too, at 92.8 using the Morisky-Green questionnaire and 94.8 based on the number of tablets missed.
Patient satisfaction and preference were also high, at approximately 90%, both in terms of how easy it was to take Binosto® and how much easier it was to take Binosto compared with other medications.
“Prof Minisola et al have conducted an interesting study. Results are interesting and needed, considering that alendronate is still a very important medication. (…) Oral bisphosphonates are considered the first line treatment in patients after fragility fractures. The present data are encouraging in terms of low prevalence of GI side effects. (…) To prescribe medications with low rate of side effects is important also during the pandemic, given the restrictions and limited access to osteoporosis clinics.”, commented one of the independent reviewers of JBMR Plus.
Overall, post-menopausal women with osteoporosis treated with Binosto® (buffered soluble alendronate) in a real-world setting experienced few upper GI AEs. In addition, they had a low discontinuation and high compliance compared to other formulations, suggesting that buffered soluble alendronate may increase patient satisfaction and therefore long-term adherence and efficacy.
“Based on my experience as the PI and first author of the pivotal trial on alendronate, a first-line treatment in osteoporosis, I believe that this study was needed and the results are relevant to provide good evidence for this alternative formulation of alendronate. The low incidence of upper GI AEs, the low discontinuation rate and the high compliance observed in the study reinforce previous clinical data regarding Binosto. These results offer confidence that Binosto could lead to increased patient satisfaction and therefore better long-term adherence and efficacy than pill forms.” commented Prof. Dennis M Black, study co-author.
1 The full paper publication can be found at: JBMR® Plus
2 World Congress on Osteoporosis, Osteoarthritis and Musculoskeletal Diseases
3 Giusti A et al. A novel effervescent formulation of oral weekly alendronate (70 mg) improves persistence compared to alendronate tablets in post-menopausal women with osteoporosis. Aging Clin Exp Res 2021; https://doi.org/10.1007/s40520-020-01777-9.
About JBMR® Plus
JBMR® Plus is the premier open access journal of the American Society for Bone and Mineral Research (ASBMR). A companion to internationally recognized title Journal of Bone and Mineral Research, JBMR® Plus aims to improve global musculoskeletal health by publishing innovative research covering endocrinology, geriatrics, orthopedics and rheumatology.
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’s go-to-market competence is proven by the development, launch and successful expansion of Binosto® in highly competitive markets.
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 for a pipeline asset.
About Binosto®
Binosto® (Buffered Soluble Alendronate) is a first-line treatment of postmenopausal osteoporosis patients that is associated with preferable pharmacologic properties translating into clinical benefit for patients. Alendronate as recommended by guidelines is a first line treatment for postmenopausal women with osteoporosis. Alendronate 70 mg tablet once weekly is the most commonly used bisphosphonate but adherence is problematic, with >50% discontinuing treatment within the first year. To address this, Binosto®, a novel buffered soluble alendronate 70 mg effervescent tablet formulation was specifically developed to improve GI tolerability and adherence to treatment.
21.10.2020
EffRx Pharmaceuticals Signs Exclusive License Agreement with Diurnal for the Registration and Commercialization of Alkindi® and Submits Market Authorization Application 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, a specialty pharmaceutical company targeting patient needs in chronic endocrine diseases, for the registration and commercialization of Alkindi® for pediatric adrenocortical insufficiency (AI) in Switzerland. Under the terms of the agreement EffRx has received the exclusive rights to register and commercialize Alkindi® in Switzerland.
Alkindi® 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 pediatric AI and the related condition congenital adrenal hyperplasia (CAH).
Alkindi® is already 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.
EffRx has recently submitted to Swissmedic (Switzerland) a Market Authorization Application for the registration of Alkindi®. Pending successful regulatory registration, this new treatment approach is expected to be available on the Swiss market by 2022. In Switzerland there are approximately 200 patients suffering from pediatric AI.
“We are extremely pleased to announce this partnership with Diurnal which demonstrates EffRx capability to expand its portfolio with promising niche and orphan medicines”, commented Lorenzo Bosisio, CEO of EffRx Pharmaceuticals. “We look forward to bringing this novel therapeutic approach to Switzerland. We are confident that Alkindi® provides a tangible advancement for young patients suffering from AI and their carers.”
Martin Whitaker, CEO of Diurnal, commented: “This partnership with EffRx further validates the quality of our products and broadens the future availability of Alkindi®. EffRx is well-placed to register and market our product Alkindi® in Switzerland. We have made strong progress with the sales of Alkindi® across Europe since its approval and subsequent launch in 2018, and we are confident this agreement will enable further growth.”
Pediatric AI, including the 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
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 for a pipeline asset.
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
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.
For further information about Diurnal, please visit www.diurnal.co.uk
Media contact: Ms. Pamela Saredi, psaredi@effrx.com
13.6.2019
EffRx Pharmaceuticals Signs Exclusive License Agreement with Pharmaxis for the Registration and Commercialization of Bronchitol® 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 entered into an exclusive license agreement with Pharmaxis Ltd, a listed pharmaceutical research company in Australia, for the registration and commercialization of Bronchitol® (mannitol) for cystic fibrosis in Switzerland.
Under the terms of the agreement, EffRx will take responsibility for registering, obtaining pricing and reimbursement as well as commercializing Bronchitol® in Switzerland. Bronchitol® works by rehydrating the airway/lung surface and promoting a productive cough, hence improving mucus clearance and lung function. The product is inhaled as dry powder twice daily using a small handheld device and it is expected to be available on the Swiss market by 2021.
Cystic fibrosis is a hereditary rare disorder affecting the exocrine glands. It causes the production of abnormally thick mucus, leading to disease manifestations primarily in the lungs, but also in pancreas, intestines, liver and kidneys. Cystic fibrosis patients often suffer from frequent respiratory tract infections. According to the Swiss Society for Cystic Fibrosis (CFCH), there are about 1,000 people affected by cystic fibrosis in Switzerland. While there is no cure for the disease, thanks to medical advances, a majority of cystic fibrosis sufferers today reach adulthood.
“We are extremely pleased to announce this exclusive licensing agreement for Bronchitol®. We look forward to making this novel therapeutic option accessible to Swiss patients suffering from cystic fibrosis”, commented Lorenzo Bosisio, CEO of EffRx Pharmaceuticals. “We are encouraged by the clinical data and experience with Bronchitol® which show that this convenient treatment is well-tolerated and can lead to improvements in lung function.”
Gary Phillips, CEO of Pharmaxis said, “Our team is delighted to have entered this collaboration with EffRx. We are keen to leverage the go-to-market expertise of EffRx to bring Bronchitol® to the Swiss market and are pleased to secure in EffRx an experienced commercial partner.”
This licensing agreement underpins EffRx strategy to expand its portfolio through licensing collaborations in Europe and Switzerland with innovative biopharmaceutical companies developing promising niche and orphan medicines.
Bronchitol® has been the subject of three large-scale, global clinical trials conducted by Pharmaxis. It is approved and marketed in Europe, Russia, Australia and several other countries. In the United States, Bronchitol® has recently received a positive recommendation from a Committee advising the US Food and Drug Administration (FDA) on the use of Bronchitol® for adult cystic fibrosis patients; with the FDA expected to advise its decision within the next few weeks.
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 aims to be the preferred accelerator for late clinical and regulatory development, approval, valorization of niche and orphan medicines in Switzerland and Europe.
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 FDA for a pipeline asset.
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 licensed in 50+ countries and marketed in the US, Europe, selected MENA and Asian countries.
About Pharmaxis
Pharmaxis (ACN 082 811 630) is an Australian pharmaceutical research company focused on inflammation and fibrosis with a portfolio of products at various stages of development and approval. Its product Bronchitol® for cystic fibrosis is marketed in Europe, Russia and Australia. Its product Aridol® for the assessment of asthma is sold in the United States, Europe, Australia and Asia. The company’s development pipeline is centered on its expertise in amine oxidase chemistry and includes a series of Lysyl Oxidase Inhibitors under clinical development targeting fibrotic diseases of the heart, kidney, liver and lung. Pharmaxis is listed on the Australian Securities Exchange (symbol PXS). The company’s head office, manufacturing and research facilities are located in Sydney, Australia. For more information about Pharmaxis, please see www.pharmaxis.com.au
Media Contact: Pamela Saredi, psaredi@effrx.com
2.4.2019
EffRx to Showcase Binosto® at the WCO-IOF-ESCEO 2019 in Paris
FREIENBACH, Switzerland–(BUSINESS WIRE)– EffRx Pharmaceuticals SA is proud to announce that it will participate in the 19th Edition of the World Congress on Osteoporosis, Osteoarthritis and Musculoskeletal Diseases, taking place April 4-7, 2019 in Paris. The WCO-IOF-ESCEO Congress is the world’s leading clinical conference on bone, joint and muscle health and therefore the ideal venue for EffRx to showcase its flagship product Binosto®, the only buffered soluble alendronate for the treatment of postmenopausal osteoporosis.
EffRx Pharmaceuticals SA is also pleased to inform that new safety data on Binosto® will be presented at the WCO-IOF-ESCEO Congress. The data will be presented as a poster titled “Upper Gastrointestinal Safety with the Buffered Solution of Alendronate 70 mg: 6 Years of Post-Marketing Experience” authored by P. Fardellone, B. Boëzennec and B. Cortet on Saturday, 6th of April 2019, from 14:00 to 15:00 (poster #579).
The authors of the new Binosto® safety data conclude that Binosto® is associated with a lower frequency of upper gastrointestinal adverse reactions than reported for alendronate tablets and that it is a well-tolerated oral bisphosphonate for the management of osteoporosis.
“These updated data reinforce the safety profile of Binosto® after 6 years of post-marketing experience,” says Dr Lorraine Zakin, Medical Affairs Director at EffRx Pharmaceuticals.
Medical professionals from around the world will be able to learn more about Binosto® and its exciting properties at the Binosto® booths 20 and 21 in the exhibition area of the Palais des Congrès de Paris.
EffRx looks forward to welcoming you to the Binosto® booth at the WCO-IOF-ESCEO Congress in Paris!
About EffRx Pharmaceuticals SA
EffRx Pharmaceuticals SA is a commercial-stage functionally integrated pharmaceutical company, based in Switzerland, that develops and commercializes niche & orphan medicines in Europe. EffRx aims to be the preferred accelerator for late clinical & regulatory development, approval, valorization of niche & orphan medicines in Europe and Switzerland.
About Binosto®/Steovess®
Our lead commercialized product, Binosto® is indicated for the treatment of osteoporosis and marketed in US, Europe and Asia. Binosto® (alendronate 70 mg effervescent tablets) is the first and only buffered solution for the treatment of postmenopausal osteoporosis. It reduces the risk of hip, spine, and non-spine fractures.1 The buffered solution with high acid-neutralizing capacity minimizes the risk of exposing the esophagus (in case of reflux) and the stomach to acidified alendronate.2,3 Binosto® is taken once a week as an easy-to-swallow, buffered solution with no risk of a tablet getting lodged in the esophagus. Binosto® has the potential to improve adherence to therapy4, thereby decreasing the risk of disabling fractures.
Disclaimer
Please refer to the official Binosto®/Steovess® prescribing information and approved indications, contraindications, and warnings in your country.
References:
- Black DM et al. Fracture Risk Reduction with Alendronate in Women with Osteoporosis: The Fracture Intervention Trial. J Clin Endocrinol Metab 2000;85:4118-4124.
- Binosto Summary of Product Characteristics. 2017.
- Hodges LA et al. Modulation of gastric pH by a buffered soluble effervescent formulation: A possible means of improving gastric tolerability of alendronate. Int J Pharm 2012;432:57-62.
- Giusti A et al. Persistence with the buffered solution of alendronate 70 mg: prospective observational study. Osteoporos Int 2018;29(S1):S431.
Media Contact: Pamela Saredi, psaredi@effrx.com