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EffRx Pharmaceuticals Signs Exclusive License Agreement with Dipharma SA and Launches Miglustat Dipharma in Switzerland

FREIENBACH, Switzerland – 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 Dipharma SA, a Swiss specialty pharmaceutical company, developing high quality, improved medicines for rare diseases. Under the terms of the agreement, EffRx has received exclusive rights to commercialize Miglustat Dipharma in Switzerland.

Miglustat Dipharma is a generic equivalent to Actelion’s (Johnson & Johnson’s) Zavesca®1. It is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is unsuitable, as well as for the treatment of progressive neurological manifestations in adult and pediatric patients with Niemann-Pick type C disease.

Gaucher disease and Niemann-Pick disease belong to a larger group of more than 50 disorders known as lysosomal storage disorders. They are inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies.

Patients with mild to moderate type 1 Gaucher disease lack an enzyme called glucocerebrosidase, which results in a glycosphingolipid called glucosylceramide building up in different parts of the body, such as the spleen, liver and bones. Miglustat Dipharma is used in patients who cannot receive the standard treatment of enzyme replacement therapy. Niemann-Pick type C is a potentially fatal disease in which glycosphingolipids build up within cells in the brain and elsewhere in the body. Miglustat Dipharma is used to treat the neurological symptoms of the disease.

The addition of Miglustat Dipharma marks another milestone for EffRx Pharmaceuticals’ growing portfolio of niche and orphan medicines in Switzerland. After having secured the rights for innovative therapies to treat conditions like cystic fibrosis and pediatric adrenal insufficiency, EffRx is thrilled to be able to launch a product which has an established place in the treatment of two major lysosomal storage disorders.

Miglustat Dipharma is reimbursed in Switzerland as of 1 November 2020.2 It provides a therapeutic alternative for patients and important cost savings for the Swiss healthcare system.

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 Dipharma SA

Dipharma S.A. is a Swiss specialty pharmaceutical company, developing high quality, improved, medicines for rare diseases. Dipharma S.A. is part of a third-generation group of family-owned companies that have grown to a global presence.

With a portfolio of generic orphan products for the treatment of Phenylketonuria, Gaucher Disease, Hereditary Tyrosinemia Type 1, Urea Cycle Disorders and others, Dipharma S.A. provides improved solutions for patients affected by inborn metabolic diseases at an affordable cost and with a global reach.

For more information, please visit www.dipharma.ch

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

1 Zavesca® is a registered trademark of Actelion Pharmaceuticals Ltd.
2 Reimbursed for the treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not possible.

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

World Osteoporosis Day – WOD marked on October 20th: THAT’S OSTEOPOROSIS

Countdown to World Osteoporosis Day:
A digital countdown checklist is an easy way to learn good bone health basics.

Get Started Now!

WOD, marked on October 20 each year, is a year-long campaign dedicated to raising global awareness of the prevention, diagnosis and treatment of osteoporosis and related musculoskeletal diseases.

It aims to put bone health and fracture prevention on the global health agenda and reaches out to health-care professionals, the media, policy makers, patients, and the public at large.

Get tested and treated if required:
  • Could you be at risk of broken bones due to osteoporosis? Take the IOF Osteoporosis Risk Check to see whether any risk factors apply to you!
  • Have you broken a bone after age 50? Without treatment, one fracture leads to another.
  • Do you have a family history of broken bones? You could be at risk.
  • Are you getting shorter? This could be a sign of spine fractures due to osteoporosis.
  • Calcium is important for bone health. Are you getting enough of this important mineral? Find out with the IOF Calcium Calculator.
  • Osteoporosis affects 1 in 3 women and 1 in 5 men aged 50+ worldwide. Don’t let it affect you! Take action for prevention.

 

EffRx Mourns Passing of Board Member Anders Wiklund

FREIENBACH, Switzerland, 19th August 2020.

The leadership of EffRx Pharmaceuticals SA is extremely saddened to announce the passing of longtime Board Member and former Chairman, Anders P. Wiklund.

Anders, a Swedish citizen, has been a board member of EffRx since its incorporation in Switzerland in 2009, and served as Chairman of EffRx between 2015 and 2016. He also acted as CEO at former EffRx Inc., USA.

“Anders will be greatly missed by the entire EffRx family. We are deeply saddened by his passing and by the loss of a friend and colleague. We are especially honored for the entrepreneurial passion towards healthcare he carried in his heart and thankful for his outstanding leadership, experience and wisdom”, said Lorenzo Bosisio, CEO of EffRx. “Anders will always remain an indelible role model in our thoughts.”

On behalf of our Board of Directors, management team and employees, we extend our deepest sympathies to Anders’ family.

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 to Showcase Binosto® at the Virtual WCO-IOF-ESCEO 2020 Congress

EffRx Pharmaceuticals SA is proud to announce that it will participate in the 20th Edition of the World Congress on Osteoporosis, Osteoarthritis and Musculoskeletal Diseases, starting on the 20th of August 2020. 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 a study on the cost-effectiveness of Binosto® will be presented at the WCO-IOF-ESCEO Congress. The data will be presented by Professor M. Hiligsmann in an oral communication (OC40) entitled, “COST-EFFECTIVENESS OF BINOSTO (BUFFERED SOLUBLE ALENDRONATE 70 MG) EFFERVESCENT TABLET FOR THE TREATMENT OF POSTMENOPAUSAL WOMEN WITH OSTEOPOROSIS IN ITALY.”

The authors of the cost-effectiveness study conclude that Binosto® represents a cost-effective strategy compared with generic alendronate, zoledronic acid and no treatment, and a dominant strategy compared to denosumab for the treatment of postmenopausal women with osteoporosis in Italy aged 60 years and over.

Medical professionals from around the world will be able to learn more about Binosto® and its exciting properties at the Binosto® virtual booth.

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.

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:

  1. 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.
  2. Binosto Summary of Product Characteristics. 2019.
  3. 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.
  4. Giusti A et al. Persistence with Buffered Solution of Alendronate 70mg: Prospective Observational Study. J Bone Miner Res 2018;33(S1):S174-S175.

EffRx Pharmaceuticals Statement on COVID-19

The globe has literally gone into quarantine due to the COVID-19 outbreak. This is an unprecedented crisis situation in terms of breadth and scale, from a global health, social and economic standpoint.

While committed to keep our core activities unaffected, EffRx has implemented measures aimed at guaranteeing business operation continuity, with particular focus on the supply of Binosto® to our customers. EffRx has engaged with its suppliers and clients to ensure continuity of the business and will continue to monitor the situation as it evolves with the aim to minimize the impact of this crisis on our business.

EffRx has further taken internal measures to reduce the contamination risk for employees and business partners. The health of our staff and partners is our number one priority. The entire staff is working remotely from home with effective equipment & systems. We have also cancelled all travels, replaced face-to-face meetings/trainings by virtual meetings and are pursuing digital collaboration with all our stakeholders.

We thank for your loyalty, flexibility and understanding for the unforeseen consequences of this global pandemic. We are in this together.

Wishing you all good health.

EffRx Pharmaceuticals
Lorenzo Bosisio
CEO

The 13th international Rare Disease Day coordinated by EURORDIS will be on February 29, 2020

Rare Disease Day takes place on the last day of February each year. The main objective of Rare Disease Day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients’ lives.

Building awareness of rare diseases is so important because 1 in 20 people will live with a rare disease at some point in their life. Despite this, there is no cure for the majority of rare diseases and many go undiagnosed. Rare Disease Day improves knowledge amongst the general public of rare diseases while encouraging researchers and decision makers to address the needs of those living with rare diseases.

Learn more about Rare Disease Day here.

Meet the EffRx Team at the 25th Annual BIO-Europe International Conference from November 11-13, 2019 in Hamburg

EffRx will be attending Europe’s largest life science partnering conference, BIO-Europe, which offers countless opportunities to engage with global life science partners. This year´s BIO-Europe partnering event will again draw over 4,000 industry attendees from over 60 countries, representing more than 2,000 companies for three days of high-level networking.

To set up a meeting, please contact us via the partnering platform or at info@effrx.com.