Vifor Pharma Group Supports Rare Disease Day 2019

The Vifor Pharma Group today announces its support of Rare Disease Day, joining patients and healthcare providers around the world to support a day that raises awareness of the impact that rare diseases have and highlights the need for a greater understanding of these debilitating conditions.

Rare Disease Day is coordinated on an international scale by the European Organisation for Rare Diseases (EURORDIS) and the National Organisation for Rare Disorders (NORD) in the US. The Rare Disease Day 2019 slogan is ‘Show Your Rare, Show You Care’ and is focused on ‘Bridging health and social care’ to address the challenges people affected by these diseases face every day. According to EURORDIS there are over 6,000 known rare diseases affecting over 300 million people worldwide.

Vifor Pharma is committed to improving the lives of patients with rare diseases around the world where high unmet medical needs exist. The company is currently developing new treatment options for three debilitating conditions:

• ANCA-associated vasculitis: ANCA-associated vasculitis is a rare, severe small vessel vasculitis affecting multiple organs with a long-term relapsing, remitting course. In partnership with ChemoCentryx, Inc., Vifor Fresenius Medical Care Renal Pharma (VFMCRP) has a product in phase-III clinical trial which expects to read out in the fourth quarter of 2019.
• Focal segmental glomerulosclerosis (FSGS): FSGS is an orphan disease of the kidney’s filtering units (glomeruli) and is characterised by serious scarring that leads to permanent kidney damage. There are currently no approved treatment options for FSGS patients. In partnership with ChemoCentryx, VFMCRP is currently developing a compound for FSGS which is currently in phase-II clinical trial.
• Beta-thalassemia: Beta-thalassemia is an inherited, rare blood disorder that reduces the production of functional haemoglobin in red blood cells, which can lead to a lack of oxygen in many parts of the body potentially causing anaemia. Severe forms can cause serious, even life-threatening complications if left untreated. Beta-thalassemia is often treated with blood transfusions which may lead to excess levels of iron in the body (iron overload). Vifor Pharma recently reported positive phase-I trial results for its oral ferroportin inhibitor and it intends to start a phase-II in the second half of 2019.

In support of Rare Disease Day, VFMCRP was a proud sponsor of the EURORDIS Black Pearl Awards – an annual event held in Brussels to recognise the outstanding achievements and exceptional work of people making a difference for the rare disease community.

“Although there has been significant progress in developing new treatments for rare diseases, there remains clear unmet need in many debilitating conditions, including ANCA-associated vasculitis where patients have to battle a severe illness, and also face difficult treatment decisions with life-long implications for their health,” said Peter Rutherford, Global Medical Lead, Orphan Renal Diseases, Vifor Pharma. “Rare Disease Day is an occasion for us at Vifor Pharma to reaffirm our strong commitment to developing new treatments and raising awareness in the conditions where we are focused.”

About the Vifor Pharma Group

The Vifor Pharma Group is a global pharmaceuticals company. It aims to become the global leader in iron deficiency, nephrology and cardio-renal therapies. The company is the partner of choice for pharmaceuticals and innovative patient-focused solutions. The Vifor Pharma Group strives to help patients around the world with severe and chronic diseases lead better, healthier lives. The company develops, manufactures and markets pharmaceutical products for precision patient care. The Vifor Pharma Group holds a leading position in all its core business activities and consists of the following companies: Vifor Pharma; VFMCRP, a joint company with Fresenius Medical Care; Relypsa; and OM Pharma. The Vifor Pharma Group is headquartered in Switzerland, and listed on the Swiss Stock Exchange (SIX Swiss Exchange, VIFN, ISIN: CH0364749348). For more information, please visit www.viforpharma.com.

About Vifor Fresenius Medical Care Renal Pharma Ltd. (VFMCRP)

Vifor Fresenius Medical Care Renal Pharma is a common company of the Vifor Pharma Group and Fresenius Medical Care, which develops and commercialises innovative and high-quality therapies to improve the lives of patients suffering from chronic kidney disease (CKD) worldwide. The company was founded at the end of 2010 and is owned 55% by Vifor Pharma Group and 45% by Fresenius Medical Care. For more information about VFMCRP and its parent companies, please visit www.vfmcrp.com, www.viforpharma.com and www.freseniusmedicalcare.com.

About EURORDIS, Rare Disease Europe

EURORDIS-Rare Diseases Europe is a unique, non-profit alliance of 826 rare disease patient organisations from 70 countries that work together to improve the lives of the 30 million people living with a rare disease in Europe. By connecting patients, families and patient groups, as well as by bringing together all stakeholders and mobilising the rare disease community, EURORDIS strengthens the patient voice and shapes research, policies and patient services. www.eurordis.org

Anti-neutrophil cytoplasmic anti-body (ANCA)-associated vasculitis

ANCA-associated vasculitis is a group of rare, relapsing/remitting, life-threatening, autoimmune diseases. ANCA-associated vasculitis can affect small blood vessels in different parts of the body resulting in damage to vital organs such as the lungs, kidneys, nervous system, gastrointestinal system, skin, eye and heart. ANCA-associated vasculitis is caused by the loss of tolerance to ANCA antigens and the development of an autoantibody, ANCA. This is a complex process involving genetic and environmental factors.^3–8

Beta-thalassemia

Beta-thalassemia is a heterogeneous autosomal recessive hereditary anaemia characterised by reduced or absent β globin chain synthesis. The resulting relative excess of unbound α globin chains precipitate in erythroid precursors in the bone marrow, leading to their premature death and, hence, to ineffective erythropoiesis. β-thalassemia phenotypes are variable, ranging from the severe transfusion dependent thalassemia major to the mild form of thalassemia intermedia.⁹ When transfusion therapy is considered, careful attention to the potential risks of iron overload should be made.¹⁰

Focal segmental glomerulosclerosis (FSGS)

Focal segmental glomerulosclerosis (FSGS) is an orphan and frequently idiopathic disease that can cause scarring in specific parts of the kidney or the filters of the organ.¹¹ FSGS is one of the causes of a serious condition known as Nephrotic Syndrome and often leads to end-stage renal disease (ESRD). There is no approved treatment option for FSGS patients.¹²

References

1. Rare Disease Day. What is Rare Disease Day? 2019. Available at: https://www.rarediseaseday.org/article/what-is-rare-disease-day. Accessed: February 2019.

2. EURORDIS Black Pearl Awards. Available at: https://blackpearl.eurordis.org/. Accessed: February 2019

3. Watts RA, et al. Nephrol Dial Transplant 2015;30(Suppl 1):i14–22.

4. Geetha D et al. J Nephrol 2015;28:17–27.

5. Hutton H et al. Semin Nephrol 2017;37:418–35.

6. Watts RA, et al. Rheumatology (Oxford) 2017;56:1439–40.

7. Robson J et al. Rheumatol Int 2018;38:675–82.

8. Jennette J and Nachman P. Clin J Am Soc Nephrol 2017;12:1680–91.

9. Danjou F, et al. Haematologica 2011;96:1573–753.

10. Taher A, et al. Guidelines for the Management of Non Transfusion Dependent Thalassemia (NTDT). 2ND EDITION Thalassemia International Federation. Publication No. 22; ISBN 978- 9963-717-11-8.

11. National Kidney Foundation. Focal Segmental Glomerulosclerosis (FSGS) 2017. Available at: https://www.kidney.org/atoz/content/focal. Accessed: February 2019.

12. Reiser J, et al. Adv Chronic Kidney Dis. 2014;21:417–21.

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Contact information

Media Relations
Heide Hauer
Head of Corporate Communications
Tel.: +41 58 851 80 87
E-mail: media@viforpharma.com