Iron deficiency

ST. GALLEN, SWITZERLAND, March 19, 2024 /PRNewswire/ -- CSL Vifor today announced that Health Canada has authorized Ferinject (ferric carboxymaltose) for the intravenous (IV) treatment of iron deficiency anemia in adult and pediatric patients one year of age and older when oral iron preparations are not tolerated or are ineffective, as well as for the treatment of iron deficiency in adult patients with heart failure and New York Heart Association (NYHA) class II/III* 1 to improve exercise capacity.2 Ferinject has now received marketing authorization in 87 countries worldwide. Research shows that rates of iron deficiency are even higher than previously reported in Canada, with the condition particularly prevalent in women of reproductive age3 and children4. "While iron deficiency and associated anemia are some of the most prevalent medical conditions on a global scale, it is easily diagnosed and treatable," said Prof. Dr. Justin Ezekowitz, Director of Cardiovascular Research and Cardiologist at the University of Alberta, Canada. "The news of a new treatment option tested in clinical trials is welcomed, given that oral irons may not be able to adequately deliver the desired outcomes for Canadian patients with heart failure impacted by iron deficiency." Iron deficiency affects one in every two hospitalized patients with heart failure5. Iron deficiency is often accompanied by reduced exercise tolerance and untreated iron deficiency may result in anemia and worsening NYHA class, with reduced quality of life and eventually increased risk of hospitalization 6,7,8 "The approval by Health Canada marks an important step forward for the treatment of iron deficiency anemia as well as iron deficiency in heart failure and supports our promise to patients and public health," said Dr. Vinicius Gomes de Lima, Head of Global Medical Affairs, CSL Vifor. "We are confident that our now approved IV iron therapy can make a meaningful contribution to achieve key therapeutic goals in the treatment of these patients." Marketing authorization in Canada is based on a comprehensive clinical data package and totality of evidence from CSL Vifor's cardiology studies. In Canada, Ferinject® is commercialized through CSL Behring Canada, Inc., with availability expected in the second half of 2024. * The New York Heart Association Functional Classification is used by Healthcare Professionals to classify patients' heart failure based on the severity of their symptoms. Patients who have a slight or marked limitation of their physical activity due to fatigue, palpitation and/or dyspnea are considered to have class II or class III heart failure, respectively.9 About CSL Vifor CSL Vifor is a global partner of choice for pharmaceuticals and innovative, leading therapies in iron deficiency and nephrology. We specialize in strategic global partnering, in-licensing and developing, manufacturing and marketing pharmaceutical products for precision healthcare, aiming to help patients around the world lead better, healthier lives. Headquartered in St. Gallen, Switzerland, CSL Vifor also includes the joint company Vifor Fresenius Medical Care Renal Pharma (with Fresenius Medical Care). The parent company, CSL (ASX: CSL; USOTC: CSLLY), headquartered in Melbourne, Australia, employs 32,000 people and delivers its lifesaving therapies to people in more than 100 countries. For more information about CSL Vifor, visit . About Ferinject Ferinject (ferric carboxymaltose) is an IV iron therapy with marketing authorization in 87 countries as of March 2024. Ferinject has historically delivered significant value to patients and healthcare systems which is based on extensive clinical data and more than 25 million patient years of exposure10. In Canada, Ferinject is indicated for the treatment of iron deficiency anemia in adult and pediatric patients one year of age and older when oral iron preparations are not tolerated or are ineffective, as well as for the treatment of iron deficiency in adult patients with heart failure and NYHA class II/III to improve exercise capacity. The diagnosis of iron deficiency must be based on laboratory tests.2 Important Information for Canada For more information and a complete risk/benefit profile, please refer to the Product Monograph available here. About iron deficiency and associated anemia Iron plays a vital role in many bodily processes, including the production of red blood cells, effective heart and brain function, and the prevention of infection and illness. Without enough iron, the body is unable to function properly. Common symptoms include fatigue, dizziness, and shortness of breath. Iron deficiency and iron deficiency anemia are estimated to affect one in three people worldwide11, yet despite the serious consequences and high prevalence12, the conditions remain under-recognized. The effects of iron deficiency differ from person to person but can be linked to an overall decline in general health and well-being13. Iron deficiency and iron deficiency anemia can be debilitating, exacerbate an underlying chronic disease and lead to increased morbidity and mortality13. Common symptoms include fatigue 13,14,15 pale skin14, brittle nails14, 16, craving non-food items such as dirt, clay and ice17, and an inability to concentrate13, 18. In children, iron deficiency anemia can significantly impair cognitive and motor development19,20,21. Multiple organizations, including the World Health Organization (WHO), UNICEF and the U.S. Agency for International Development (USAID) have called for a comprehensive anemia monitoring framework. Find more information on the global burden of anemia in the report published in the Lancet in July 2023: . References Fox AC, Gorlin R, Levin RI, New York Heart Association. Criteria Committee. Nomenclature and criteria for diagnosis of diseases of the heart and great vessels. 9th ed. Boston, MA: Lippincott Williams and Wilkins; March 1, 1994. Ferinject Product Monograph. CSL Vifor, St. Gallen, Switzerland. Chang V et al. J Nutr 2022 ; 153 : 781–797. doi.org/10.1016/j.tjnut.2022.10.011; Cochrane K et al. J Nutr 2022;152:2238–2244. Tahir E et al. 2020 Can J Public Health 111, 682–693 (2020). doi.org/10.17269/s41997-020-00304-7; Abdullah K et al Canadian Paediatric Surveillance program, 2011 . Del Pinto et al. Eur Heart J Suppl. 2022 Nov; 24(Suppl I): I96–I99). Ebner N, von Haehling S. Iron deficiency in heart failure: a practical guide. Nutrients. 2013;5(9):3730-9. doi:10.3390/nu5093730. Klip IT, Comin-Colet J, Voors A a, et al. Iron deficiency in chronic heart failure: an international pooled analysis. Am Heart J. 2013;165(4):575-582.e3. doi:10.1016/j.ahj.2013.01.017. Guedes M NDT 2021. doi: 10.1093/ndt/gfab050; Enjuanes C, et al. Int J Cardiol. 2014;174(2):268–275. Classes of Heart Failure. American Heart Association. May 2017. Data on file. PSUR report. CSL Vifor, St. Gallen, Switzerland. Peyrin-Biroulet L, et al. Guidelines on the diagnosis and treatment of iron deficiency across indications: a systematic review. Am J Clin Nutr. 2015;102(6):1585-94. World Health Organization. Worldwide prevalence of anaemia 1993-2005. 2008. Available at URL: E0C7?sequence=1. Last accessed: November 2023. Fernando B, et al. A guide to diagnosis of iron deficiency and iron deficiency anemia in digestive diseases. World J Gastroenterol. 2009 Oct 7; 15(37): 4638-4643. Auerbach M, Adamson JW. How we diagnose and treat iron deficiency anemia. Am J Hematol. 2016;91(1):31-38. Favrat, B., et al. (2014). Evaluation of a single dose of ferric carboxymaltose in fatigued, iron-deficient women--PREFER a randomized, placebo-controlled study. PLoS One 9(4): e94217. eCollection 2014. Cashman MW, Sloan SB. Nutrition and nail disease. Clin Dermatol. 2010;28(4):420-5. Barton JC, et al. Pica associated with iron deficiency or depletion: clinical and laboratory correlates in 262 non-pregnant adult outpatients. BMC Blood Disord. 2010;10:9. doi:10.1186/1471-2326-10-9. Patterson A et al. Iron deficiency, general health and fatigue: Results from the Australian Longitudinal Study on Women's Health. Qual Life Res. 2000;9:491-497. World Health Organization. Nutritional anaemias: tools for effective prevention and control. 2017. Available at URL: . Last accessed: November 2023. Sant-Rayn S, et al. Diagnosis and management of iron deficiency anaemia: a clinical update. Med J Aust 2010; 193 (9): 525-532. doi: 10.5694/j.1326-5377.2010.tb04038.x. Özdemir N. Iron deficiency anemia from diagnosis to treatment in children. Turk Pediatri Ars. 2015 Mar 1;50(1):11-9. doi: 10.5152/tpa.2015.2337. PMID: 26078692; PMCID: PMC4462328. CSL Vifor Media Contact Thomas Hutter +41 79 957 96 73 [email protected] SOURCE Vifor International AG (CSL Vifor)

BRONX, N.Y., March 8, 2024 /PRNewswire/ -- As we age, our hematopoietic (blood-forming) stem cells (HSCs) become less able to produce new red and white blood cells and other vital blood components—contributing to chronic inflammation and accelerating the onset of blood cancers and degenerative diseases. Researchers at Albert Einstein College of Medicine and the National Cancer Institute-designated Montefiore Einstein Comprehensive Cancer Center (MECCC) have found that failing HSC function can be prevented or even reversed by reducing iron levels in these adult stem cells. The findings published March 7 in the print edition of the journal Cell Stem Cell. Continue Reading Britta Will, Ph.D., associate professor of oncology, of medicine, of cell biology "We've shown that this decline in HSC function is not inevitable and appears to be reversible," said study senior and co-corresponding author Britta Will, Ph.D., associate professor of oncology, of medicine, of cell biology, and the Belfer Scholar in Cancer Research at Einstein, and co-leader of the stem cell & cancer biology program at MECCC. "By lowering iron levels inside the cell with a clinically available drug, we were able to restore a youthful pool of HSCs in older mice. This simple treatment strategy holds promise for slowing aging-related diseases as well as chronic inflammatory diseases and blood cancers." An Unexpected Role for Iron in Blood Stem Cells This research dates to 2018, when Dr. Will and colleagues observed that eltrombopag, a drug used to boost levels of platelet blood cells, also stimulates the activity of HSCs. At that time, the researchers discovered that this improvement occurs because eltrombopag removes, or chelates, iron from HSCs. Their new study showed that the amount of iron inside HSCs determines the cells' fate and function. After analyzing HSCs, the researchers found that excess intracellular iron activates inflammation within HSCs and pushes them towards dormancy—a state in which a cell's functions are massively slowed, similar to animal hibernation. Dormancy limits HSCs' ability to make more copies of themselves and to produce sufficient numbers of high-quality blood cells. Conversely, HSCs with restricted iron levels readily multiply and respond effectively when more blood components are needed. "So basically, iron restriction governs and protects the regenerative capacity of stem cells—their ability to divide and to differentiate into blood cells," said the paper's co-corresponding author Yun-Ruei Kao, Ph.D., research assistant professor of oncology and of medicine at Einstein. By what mechanism does iron limitation maintain HSC health? In studies involving young mice, the researchers found that low iron triggers a molecular response in HSCs that temporarily increases fatty acid metabolism, strengthening genetic programs in HSCs after they've proliferated in young animals. By contrast, HSC in aged mice were found to contain increased iron levels that inhibited the activation of this newly discovered fatty-acid metabolism pathway. The researchers next studied whether removing iron from HSCs would improve blood-cell production in old and aging mice. To reduce iron levels in HSCs, the team injected old mice with the iron chelator deferoxamine daily for 14 days. Compared with controls, aged HSCs exposed to iron chelation produced significantly more blood cells after their HSCs were transplanted into other mice. Longer-term iron chelation had an even more pronounced effect: When 6-month-old (middle-aged) mice were given an iron chelator for 13 additional months (well into old age), the animals' HSCs showed up to a 10-fold increase in regenerative capacity compared with controls. Aging and Iron Intake Since aging is widely associated with systemic iron deficiency, doctors often advise seniors to take iron supplements or eat more iron-rich foods. This supplemental iron lowers the risk of iron deficiency anemia, the source of numerous health issues. Those recommendations still hold, Dr. Will said. She noted that iron metabolism can deteriorate during aging, leading to iron deficiency in some organs and iron loading in others, including in the bone marrow where many HSCs are located. "Our study's key finding—that chelators, by neutralizing iron loading inside HSCs, may enable HSCs to remain healthy into old age—is potentially important," said Dr. Will. "To our knowledge, this is the first time that a non-invasive strategy has successfully rejuvenated stem cells. But like all experimental strategies, it needs to be assessed in a clinical setting to make sure it's safe and effective." Dr. Will is currently exploring HSCs and iron from several angles: how to safely limit iron in HSCs during aging; whether restricting iron levels within HSCs can prevent their aging-associated transformation into cancerous HSCs; and to what extent other adult stem cells, such as those in the gut, depend on iron restriction to reliably regenerate tissues and organs. The Cell Stem Cell paper is titled "An iron rheostat controls hematopoietic stem cell fate." In addition to Dr. Kao, Jiahao Chen, Ph.D., is co-first author of the study. Other Einstein authors include Rajni Kumari, Ph.D., Aliona Zintiridou, Madhuri Tatiparthy, M.Sc., Yuhong Ma, Ph.D., Maria M. Aivalioti, Ph.D., Deeposree Moulik, B.Sc., Sriram Sundaravel, Ph.D., Daqian Sun, Ph.D., Juliane Grimm, M.D., Stephanie Stransky, Ph.D., Simone Sidoli, Ph.D., and Ulrich Steidl, M.D., Ph.D. Additional authors are Julie Reisz, Ph.D., and Angelo D'Alessandro, Ph.D., at the University of Colorado; as well as Rajat Singh, M.D., and Nuria Martinez-Lopez, Ph.D., at the University of California Los Angeles. Drs. Will and Steidl have received funds for research projects and for serving on the advisory board of Novartis Pharmaceuticals. SOURCE Albert Einstein College of Medicine

SUMMIT, N.J., March 6, 2024 /PRNewswire/ -- CSL Seqirus, a global leader in the protection of public health, confirmed it is fully prepared to deliver its influenza vaccine portfolio for the 2024/25 U.S. season, based on the trivalent strains recommended this week by the U.S. Food and Drug Administration's (FDA) Vaccines and Related Biological Products Advisory Committee (VRBPAC). During the meeting, the committee confirmed the viral strain selection of influenza vaccines for the Northern Hemisphere 2024/25 season, which aligns with the February 2024 World Health Organization (WHO) annual recommendations and is unchanged from the Southern Hemisphere 2024 season.1 "CSL Seqirus applauds the VRBPAC's commitment to providing a clear, evidence-backed recommendation to the FDA so that the composition of seasonal influenza vaccines for the upcoming season will be formulated to match projected circulating strains, based on the WHO's ongoing surveillance," said Gregg Sylvester, Chief Health Officer and Head of Medical Affairs, CSL Seqirus. "In addition to implementing this change, we are committed to working with our partners in public health to reverse the alarming decline in immunization rates and vaccine confidence that we have seen over the past two flu seasons." The VRBPAC's recommendation follows the October 2023 VRBPAC meeting, where the committee strongly supported the WHO's recommendation to expeditiously remove the B/Yamagata influenza virus strain from quadrivalent influenza vaccines and transition to trivalent influenza vaccines for the 2024/25 season.2 The recommendation was grounded in data from the WHO's global influenza response and surveillance system, which detected a decline in B/Yamagata circulation before the onset of the COVID-19 pandemic and no further circulation of B/Yamagata lineage viruses since March of 2020.3 The strain selection for the 2024/25 influenza season reflects the removal of B/Yamagata and it will not be included in the vaccines manufactured and delivered by CSL Seqirus. CSL Seqirus worked closely with the FDA during 2023 to align on a path forward and is fully prepared to transition its complete portfolio of seasonal influenza vaccines in the U.S. market from quadrivalent to trivalent formulations for the 2024/25 influenza season. CSL Seqirus completed its transition plan for the 2024/2025 season by December 2023 and regulatory and manufacturing teams are on track to convert the full U.S. portfolio. As a result of this effort, CSL Seqirus received FDA approval on March 4, 2024 for all of its U.S. trivalent influenza vaccines. "This strain consistency across hemispheres will support both speed and efficiency, as CSL Seqirus has experience manufacturing these recommended strains," said Dave Ross, Vice President, North America Commercial Operations, CSL Seqirus. Across the world, CSL Seqirus is collaborating with regulatory bodies and public health authorities on an appropriate transition timeline for each country that aims to ensure a smooth transition, increase vaccine confidence and improve immunization rates. The undetectable circulation of B/Yamagata is a testament to the critical role of widely implemented influenza immunization programs, amongst other contributing factors such as the inherent characteristics and epidemiology of the B/Yamagata virus and the unique environment created by the COVID-19 pandemic.3 About VRBPAC The VRBPAC is the FDA's advisory committee of experts in the fields of immunology, molecular biology, rDNA, virology bacteriology, epidemiology or biostatistics, vaccine policy, vaccine safety science, federal immunization activities, vaccine development including translational and clinical evaluation programs, allergy, preventive medicine, infectious diseases, pediatrics, microbiology, and biochemistry.4 VRBPAC reviews and evaluates data concerning the safety, effectiveness, and appropriate use of vaccines and related biological products which are intended for use in the prevention, treatment, or diagnosis of human diseases, and, as required, any other products for which the FDA has regulatory responsibility.4 About Seasonal Influenza Influenza is a common, contagious seasonal respiratory disease that may cause severe illness and life-threatening complications in some people.5 Influenza can lead to clinical symptoms varying from mild to moderate respiratory illness to severe complications, hospitalization and in some cases, death. Because transmission of influenza viruses to others may occur one day before symptoms develop and up to 5 to 7 days after becoming sick, the disease can be easily transmitted to others.5 Preliminary estimates from the Centers for Disease Control and Prevention (CDC) report that during the 2023/24 influenza season, there have been an estimated 290,000-610,000 influenza-related hospitalizations in the U.S.6 The CDC recommends annual vaccination for individuals aged 6 months and older, who do not have any contraindications.7 Since it takes about two weeks after vaccination for antibodies to develop in the body that help protect against influenza virus infection, it is recommended that people get vaccinated before influenza begins spreading in their community.7 The CDC recommends that for most people who need only 1 dose of flu vaccine, vaccination should ideally happen in September or October.7 About CSL Seqirus CSL Seqirus is part of CSL (ASX: CSL). As one of the largest influenza vaccine providers in the world, CSL Seqirus is a major contributor to the prevention of influenza globally and a transcontinental partner in pandemic preparedness. With state-of-the-art production facilities in the U.S., the U.K. and Australia, and leading R&D capabilities, CSL Seqirus utilizes egg, cell and adjuvant technologies to offer a broad portfolio of differentiated influenza vaccines in more than 20 countries around the world. For more information about CSL Seqirus, visit CSL.com/CSLSeqirus. About CSL CSL (ASX: CSL) (USOTC: CSLLY) is a global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat haemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our three businesses: CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 32,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For inspiring stories about the promise of biotechnology, visit CSL.com/Vita and follow us on Twitter.com/CSL. For more information about CSL, visit . Intended Audience This press release is issued from CSL Seqirus in Summit, New Jersey, USA and is intended to provide information about our global business. Please be aware that information relating to the approval status and labels of approved CSL Seqirus products may vary from country to country. Please consult your local regulatory authority on the approval status of CSL Seqirus products. Forward-Looking Statements This press release may contain forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause our actual results, performance or achievements to be materially different from any future results, performances or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. USA-CRP-24-0011 MEDIA CONTACT Tiffany Cody +1 (908) 370-1863 [email protected] REFERENCES 1 Food and Drug Administration (FDA). Center for Biologics Evaluation and Research. 184th Vaccines and Related Biological Products Advisory Committee. March 2024. Retrieved from: . Accessed March 2024. 2 FDA. Center for Biologics Evaluation and Research. 183rd Vaccines and Related Biological Products Advisory Committee . Challenges and Opportunities for Vaccine Strain Composition with the Reduced Public Health Threat from Influenza B/Yamagata Lineage Viruses. Slide 7. October 2023. Retrieved from: . Accessed March 2024. 3 Koutsakos, M., Wheatley, A. K., Laurie, K., Kent, S. J., & Rockman, S. (2021). Influenza lineage extinction during the COVID-19 pandemic? Nature Reviews Microbiology, 19(12), 741-742. . 4 FDA. Center for Biologics Evaluation and Research. Vaccines and Related Biological Products Advisory Committee . (2019). Retrieved from: . Accessed March 2024. 5 Centers for Disease Control and Prevention (CDC). (2022). Key Facts About Influenza (Flu). Retrieved from: . Accessed March 2024. 6 CDC. (2024). 2023-2024 U.S. Flu Season: Preliminary In-Season Burden Estimates. Retrieved from: . Accessed March 2024. 7 CDC. (2024). Who Needs a Flu Vaccine. Retrieved from: . Accessed March 2024. SOURCE CSL Seqirus