Rickets

The XLH Network and Kyowa Kirin publish findings from community survey, accompanying white paper New evidence shows wide-ranging impact of XLH, including finances, daily activities, and mental health Survey insights will be shared at the NORD Breakthrough Summit in Washington, D.C., October 20-22 PRINCETON, N.J., Oct. 15, 2024 /PRNewswire/ -- Kyowa Kirin, Inc., a wholly owned subsidiary of Kyowa Kirin Co. Ltd., along with The XLH Network, a non-profit patient advocacy organization, today announced the publication of Insights for Change: Findings from the XLH Community Impact Survey, an inside look at the experiences of people living with X-linked hypophosphatemia (XLH), a rare, progressive, genetic disease that impacts the bones and muscles in both children and adults.1 Continue Reading XLH joint logo View PDF The white paper reveals the broader impact of XLH on U.S. patients and their families, including personal finances and out of pocket expenses; physical, mental, and social health; and the challenges faced when accessing expert care. The full survey data report and white paper are the result of a collaboration between Kyowa Kirin and The XLH Network and are now available along with other resources at XLHimpact.com. "For generations, families living with XLH have struggled to explain the disease to many of the professionals involved in their care. Without the data to back them up, patients have often reported feeling dismissed, or that their concerns were not validated," said Susan Faitos, Executive Director of The XLH Network. "Research projects like this, which focus on the actual lived experience of someone with XLH, will lead to a greater understanding of XLH and facilitate meaningful discussions between XLHers and all those involved in their care. The XLH Network is proud to be a part of this project." More than 85 adults with XLH and caregivers of children with XLH responded to the online survey, and 56 of these respondents completed additional follow-up phone interviews. The following key findings illustrate the ongoing challenges of living with XLH. Access to Care: XLHers face unique challenges accessing care. Survey respondents rated their XLH doctors as fairly knowledgeable; however, they spent considerable time and money to access their doctors. Survey respondents reported traveling on average 180 miles roundtrip, taking over three hours just to get to and from their XLH doctor's office. These travel requirements proved to be particularly costly, amounting to on average $1,000 and as much as $15,000 in annual travel costs for survey respondents. Financial Burden: The broad health implications of living with XLH are reflected in the wide array of expenses reported for direct XLH care and other expenses such as home modifications, dental procedures, and travel to doctor offices. Survey respondents indicated that they spent an average of 6% – and in one case as much as 40% – of their annual household income on out of pocket (OOP) expenses related to managing or living with XLH in the last year. In addition, over the past year, 61% of respondents paid OOP for dental care, with annual costs averaging $2,775. Impact on Daily Activities: The survey and white paper also document the considerable toll XLH has on an individuals' day-to-day and social life. When asked to identify the most burdensome impacts on their daily lives, XLHers commonly cited absenteeism, reduced productivity at work or school, and challenges with personal relationships. Some adult survey respondents noted that XLH restricts daily activities such as getting dressed, housework, driving, shopping, and cooking. Mental Health Impact: XLH can strain patients' and families' mental and emotional health resources. A majority of respondents – 83% of adults and 90% of children – reported at least one mental health issue related to their XLH. While most expressed concerns about their overall mental health, very few said they were seeing a mental health professional to address these concerns (13% of adults, 3% of children). Transition of Care: Because of the lifelong, progressive nature of XLH, most XLHers are diagnosed during childhood but continue to experience symptoms into adulthood.1 Nevertheless, an overwhelming majority (94%) of adult XLHers who completed the survey reported they did not formally transition their care from a pediatrician to an adult healthcare professional (HCP). When asked for a reason, 14 of the 49 adults who were diagnosed before age 18, stated they were advised the disease "would go away over time." "Kyowa Kirin is deeply committed to listening to and working with the XLH community to advance the understanding of XLH and the challenges this disease places on patients and their families," said Lauren Walrath, Vice President of Public Affairs at Kyowa Kirin North America. "As this survey makes clear, the needs of every XLHer are unique and while progress has been made, more work is needed to make care accessible and to ease the burden on families. We look forward to working with The XLH Network, healthcare providers, and other stakeholders on solutions that provide meaningful change at the community level." About the XLH Community Impact Survey The XLH Community Impact Survey is a collaboration between Kyowa Kirin and The XLH Network. The purpose of the survey was to quantify and deepen understanding of the lived experiences of adults, caregivers, and children with XLH in the United States. The survey evaluated the impact of XLH on personal finances and physical, mental, and social health, as well as access to expert quality care. Between November 2023 and February 2024, a total of 87 adults living with XLH and caregivers of children with XLH completed an online survey, with follow-up phone interviews conducted with 56 of the respondents. Survey responses reflect respondent variation across key demographics such as age, geography, and insurance status, and may not reflect the experiences of all individuals with XLH. Adult males, young adults aged 18-44, and individuals from rural settings were under-represented among respondents. Engage Health designed, fielded, and conducted analysis of the survey with analytic and editorial support from SmithSolve and input from The XLH Network and Kyowa Kirin, Inc. To learn more about XLH and the survey results, and to access additional resources, visit XLHimpact.com. X-linked hypophosphatemia X-linked hypophosphatemia is a rare, lifelong, genetic disease that can impact the bones and muscles in both children and adults.1 In individuals with XLH, the body doesn't hold on to enough phosphorus, which is an essential mineral for bone health.1 This is due to the production of excess fibroblast growth factor 23 (FGF23), causing the body to release too much phosphorus through the urine.1 When phosphorus levels are too low (hypophosphatemia), it can cause the softening and weakening of growing bones in children (rickets) and of mature bones in children and adults (osteomalacia).1 In children, XLH typically appears as bowed legs or knock knees.1 Over time, bone weakening can also lead to bone abnormalities in the legs, delayed growth, and short stature.1 In adults, XLH may cause osteomalacia, fractures and pseudo-fractures, and hypophosphatemia.1 About Kyowa Kirin Kyowa Kirin aims to discover novel medicines with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, we have invested in drug discovery and biotechnology innovation for more than 70 years and are currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients affected by severe and rare diseases. A shared commitment to our values, to sustainable growth, and to making people smile unites us across Japan, North America, and EMEA/International. You can learn more about Kyowa Kirin North America at: kkna.kyowakirin.com or LinkedIn: Kyowa Kirin Inc. U.S. About The XLH Network The XLH Network supports families and individuals living with X-Linked Hypophosphatemia (XLH). Our mission includes promoting awareness and education, providing opportunities for connection and community, and to foster the search for a cure. Our vision is early, accurate diagnosis; readily available, well-managed treatment; and healthy quality of life for those who are affected with XLH and related disorders. COR-US-CRY-0096 October 2024 1Dahir K, et al. X-linked hypophosphatemia: a new era in management. J Endocr Soc. 2020;4(12):bvaa151. SOURCE Kyowa Kirin WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

- Data underscore the urgent need for therapies that address the cardiovascular and musculoskeletal complications and long-term systemic effects of ENPP1 and ABCC6 Deficiencies in children - - Inozyme and GACI Global launched PROPEL Registry to further understanding of these rare, life-threatening diseases (NCT06302439) - BOSTON, Sept. 26, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage biopharmaceutical company developing innovative therapeutics for rare diseases that affect bone health and blood vessel function, is presenting four posters at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting, including new data that demonstrate the progression and impact of rare bone and blood vessel diseases in children. These presentations include findings highlighting the significant medical burden associated with ENPP1 Deficiency and early-onset ABCC6 Deficiency, new data from a natural history study that provides critical insights into the progression of ENPP1 Deficiency, and the launch of a global registry to enhance our understanding of these diseases. “Given their high mortality rate, there is an urgent unmet need for patients affected by these rare bone and blood vessel diseases. For instance, more than half of all infants with ENPP1 Deficiency die within six months, and those that survive often face major and potentially fatal complications throughout their lives,” said Kurt Gunter, M.D., Chief Medical Officer of Inozyme Pharma. “These new data underscore the significant and multifaceted impact of ENPP1 and ABCC6 Deficiencies, further highlighting the significant need for therapies that address the immediate and long-term systemic complications associated with these conditions. At Inozyme, we are dedicated to unlocking the full potential of our lead candidate, INZ-701, a potentially transformative treatment for patients and their families facing these diseases.” “As the data from these retrospective analyses demonstrates, the impact of these life-threatening conditions is more significant in both the short and long-term than we had understood, particularly in terms of cardiovascular and musculoskeletal complications,” said Christine O’Brien, co-president of GACI Global. “We’re excited to work with Inozyme to launch the PROPEL Registry to prospectively collect clinical data and patient-reported outcomes that will provide a comprehensive understanding of the burden of illness and progressive nature of ENPP1 Deficiency and early-onset ABCC6 Deficiency.” Natural History Study of ENPP1 Deficiency ENPP1 Deficiency is a serious and progressive rare disease that affects blood vessels, soft tissues, and bones. Individuals who present in utero or in infancy are typically diagnosed with generalized arterial calcification of infancy (GACI Type 1), with about 50% of these infants not surviving beyond six months. Key findings of the retrospective analysis included: In the 84 patients with ENPP1 Deficiency, cardiovascular complications and arterial calcifications developed early in life, predominately within the first few months.A striking 88% of patients experienced ectopic calcifications, with 81% showing arterial calcification and 66.7% showing aortic calcification. Cardiovascular complications, including hypertension, heart valve defects and heart failure, were noted in 76% of patients.Among the 84-patient cohort, 51 were diagnosed with Generalized Arterial Calcification of Infancy (GACI) alone and only 19 survived beyond infancy; 22 were diagnosed with GACI, survived beyond infancy and progressed to Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2); and 11 patients presented initially with ARHR2.Musculoskeletal complications, including phosphate-wasting rickets, bowed extremities, and pain, were documented in all patients diagnosed with ARHR2, with 45% of ENPP1 Deficiency survivors showing symptoms by age 3 and 75% by age 11.The data highlight that 95% of surviving ENPP1 Deficiency patients are expected to experience cardiovascular, musculoskeletal, and other organ complications, underscoring the lifelong burden of the disease. Title: Lifetime Accumulation of Calcification and Clinical Complications in Patients with ENPP1 Deficiency (GACI and ARHR2)Format: Poster PresentationAuthor/Presenter: Carlos Ferreira, M.D., of the National Institutes of Health (NIH)Session Dates and Times: Rare Bone Disease Symposium: Thursday, September 26, 2024, 8:00 a.m. – 6:00 p.m. ET Early Career Spotlight: Friday, September 27, 2024, 4:54 p.m. ET Plenary during Welcome Reception: Friday, September 27, 2024, 5:30 – 7:30 p.m. ET Poster Session I: Saturday, September 28, 2024, 2:15 – 3:45 p.m. ET Retrospective Observational Study of ENPP1 Deficiency and Early-onset ABCC6 Deficiency Like ENPP1 Deficiency, ABCC6 Deficiency is a progressive and debilitating rare disease that affects blood vessels and soft tissues. Infants with ABCC6 Deficiency are diagnosed with generalized arterial calcification of infancy (GACI Type 2), which is similar to GACI Type 1, the infant form of ENPP1 Deficiency. This study described the clinical events and interventions in patients with ENPP1 Deficiency (n=14) and early-onset ABCC6 Deficiency (n=9).ENPP1 Deficiency was associated with a high burden of cardiovascular complications in infancy, with additional frequent medical interventions including orthopedic surgery (64%) and hearing aids (43%).Early-onset ABCC6 Deficiency patients showed significant cardiovascular complications, and 44% experienced stroke. Title: The Medical Burden of ENPP1 Deficiency and Early-Onset ABCC6 Deficiency from a Retrospective Observational StudyFormat: Poster PresentationAuthor/Presenter: David R. Weber, M.D., MSCE, Attending Physician and Medical Director of the Center for Bone Health at Children’s Hospital of PhiladelphiaSession Dates and Times: Rare Bone Disease Symposium: Thursday, September 26, 2024, 8:00 a.m. – 6:00 p.m. ET Plenary during Welcome Reception: Friday, September 27, 2024, 5:30 – 7:30 p.m. ET Poster Session I: Saturday, September 28, 2024, 2:15 – 3:45 p.m. ET Additional poster presentations include: Title: PROPEL: Prospective Observational Registry for ENPP1 Deficiency and Infantile-Onset ABCC6 DeficiencyFormat: Poster PresentationAuthor/Presenter: Lothar Seefried, M.D., of the Orthopedic Department of the University Hospital at the Julius-Maximilians University Würzburg, GermanySession Dates and Times: Energy Metabolism in Skeletal Development and Disease: Thursday, September 26, 2024, 8:30 a.m. – 5:30 p.m. ET Poster Session I: Saturday, September 28, 2024, 2:15 – 3:45 p.m. ET Title: Therapeutic Application of ENPP1-Fc Prevent Muscle Calcification Following Cardiotoxin-induced-muscle Damage in Abcc6 -/- miceFormat: Poster PresentationAuthor/Presenter: Kevin O’Brien, Principal Scientist, Inozyme PharmaSession Date and Time: Late Breaking Poster Session I: Saturday, September 28, 2024, 2:15 – 3:45 p.m. ET About ENPP1 Deficiency ENPP1 Deficiency is a serious and progressive rare disease that affects blood vessels, soft tissues, and bones. Individuals who present in utero or in infancy are typically diagnosed with generalized arterial calcification of infancy (GACI Type 1), with about 50% of these infants not surviving beyond six months. Children with this condition typically develop rickets, specifically autosomal-recessive hypophosphatemic rickets type 2 (ARHR2), while adolescents and adults may develop osteomalacia, or softened bones. ARHR2 and osteomalacia cause pain and difficulty with movement. Additionally, patients may experience hearing loss, calcification in arteries and joints, and heart problems. Biallelic ENPP1 Deficiency affects approximately 1 in 64,000 pregnancies worldwide. Initially, it was believed to only impact individuals with two copies of the mutated gene. However, many individuals with just one copy of the mutated gene (monoallelic ENPP1 Deficiency) also exhibit severe symptoms. This suggests that the worldwide prevalence of ENPP1 Deficiency may be much higher than current estimates, which are based solely on biallelic cases. Currently, there are no approved therapies for ENPP1 Deficiency. About ABCC6 Deficiency ABCC6 Deficiency is a progressive and debilitating rare disease that affects blood vessels and soft tissues. Infants with ABCC6 Deficiency are diagnosed with generalized arterial calcification of infancy (GACI Type 2), which is similar to GACI Type 1, the infant form of ENPP1 Deficiency. Pediatric patients who survive beyond the first year of life may develop neurological disease, including strokes, and cardiovascular diseases due to ongoing vascular calcification and stenosis. In older individuals, ABCC6 Deficiency manifests as pseudoxanthoma elasticum (PXE), characterized by abnormal mineralization in blood vessels and soft tissues, affecting the skin, visual function, and vascular system. Biallelic ABCC6 Deficiency is estimated to affect 1 in 25,000 to 1 in 50,000 individuals worldwide. Initially, it was believed to only impact individuals with two copies of the mutated gene. However, many people with just one copy of the mutated gene (monoallelic ABCC6 Deficiency) also exhibit severe symptoms. This suggests that the worldwide prevalence of ABCC6 Deficiency may be much higher than current estimates, which are based solely on biallelic cases. Currently, there are no approved therapies for ABCC6 Deficiency. About Inozyme PharmaInozyme Pharma is a pioneering clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics for rare diseases that affect bone health and blood vessel function. We are experts in the PPi-Adenosine Pathway, where the ENPP1 enzyme generates inorganic pyrophosphate (PPi), which regulates mineralization, and adenosine, which controls intimal proliferation (the overgrowth of smooth muscle cells inside blood vessels). Disruptions in this pathway impact the levels of these molecules, leading to severe musculoskeletal, cardiovascular, and neurological conditions, including ENPP1 Deficiency, ABCC6 Deficiency, calciphylaxis, and ossification of the posterior longitudinal ligament (OPLL). Our lead candidate, INZ-701, is an ENPP1 Fc fusion protein enzyme replacement therapy (ERT) designed to increase PPi and adenosine, enabling the potential treatment of multiple diseases caused by deficiencies in these molecules. It is currently in clinical development for the treatment of ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis. By targeting the PPi-Adenosine Pathway, INZ-701 aims to correct pathological mineralization and intimal proliferation, addressing the significant morbidity and mortality in these devastating diseases. For more information, please visit https://www.inozyme.com/ or follow Inozyme on LinkedIn, X, and Facebook. Cautionary Note Regarding Forward-Looking StatementsStatements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the potential benefits of INZ-701. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to conduct its ongoing clinical trials of INZ-701 for ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis; enroll patients in ongoing and planned trials; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain, and protect intellectual property rights related to its product candidates; manage expenses; comply with covenants under its outstanding loan agreement; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section in the Company's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. Contacts Investors:Inozyme PharmaStefan Riley, Senior Director of IR and Corporate Communications(857) 330-8871Stefan.riley@inozyme.com Media:Biongage CommunicationsTodd Cooper(617) 840-1637Todd@biongage.com

One oral presentation and nine posters will shed light on real-world experiences of people living with XLH and the impact of burosumab treatment TOKYO, Sept. 19, 2024 /PRNewswire/ -- Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE:4151) will share new research that enhances understanding about the use of burosumab in real-world clinical practice as well as the burden of those living with X-linked hypophosphatemia (XLH), a rare genetic metabolic bone disease. The findings will be presented at the American Society for Bone and Mineral Research (ASBMR) 2024 annual meeting, which takes place September 27-30 in Toronto. The oral presentation of "Patient reported outcomes (PRO) from a real-world study of burosumab treatment in adults with X-linked hypophosphatemia in the UK" will be made by Judith Bubbear, MD, Royal National Orthopaedic Hospital (UK) on Saturday, September 28, 11:30 am EDT (Presentation #1022). "As we learn more about XLH and how it impacts people's lives, real-world evidence can provide much-needed insight into potential strategies for managing this debilitating disease," said Angela Williams, PhD, Vice President and Global Head of Health Economics and Outcomes Research at Kyowa Kirin. "The breadth of our ASBMR data reflects our continued focus on advancing the science and understanding of XLH and improving the lives of people living with XLH." ASBMR XLH poster presentations: Real-world impact of burosumab treatment in people living with XLH: "Effectiveness of burosumab versus conventional therapy in adults with X-linked hypophosphatemia in a real-world setting from the XLH Disease Monitoring Program" (Poster#SAT-430) Lead author: Pablo Florenzano, MD, Pontificia Universidad Católica de Chile Poster Session I: Saturday, September 28, 2:15 – 3:45 pm EDT "Biochemical measurements according to age group in burosumab-treated patients with X-linked hypophosphatemia (XLH) in a real-world setting: an analysis of the XLH Disease Monitoring Program" (Poster #Fri-424, #Sun-424) Lead author: Leanne M. Ward, MD, Children's Hospital of Eastern Ontario Welcome Reception/Plenary Poster Session: Friday, Sept. 27, 5:30 – 7:30 pm EDT Poster Session II; Sunday, Sept. 29, 2:15 – 3:45 pm EDT "Symptoms experienced by adolescents living with X-linked hypophosphatemia at the end of skeletal growth (EOSG) treated with burosumab—a mixed methods analysis" (Poster #SAT-445) Lead author: Vrinda Saraff, MD, Birmingham Women's and Children's Hospital (UK) Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT "Efficacy and safety of burosumab in adult Chinese patients with X-linked hypophosphatemic rickets/osteomalacia (XLH): an open-label, multi-center, single-cohort, post-marketing phase IV study" (Poster#SUN-119) Lead author: Wei Liu, MD, Peking Union Medical College Hospital Poster Session II: Sunday, Sept. 29, 2:15 – 3:45 pm EDT "Efficacy and safety of burosumab in pediatric Chinese patients with X-linked hypophosphatemic rickets/osteomalacia (XLH): an open-label, multi-center, single-cohort, post-marketing phase IV study" (Poster#SAT-121) Lead author: Xiaoping Luo, MD, Tongji Medical College, Huazhong University of Science and Technology Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT XLH burden on multiple aspects of patients' lives: "Association between pain medication use and patient-reported outcomes in adults with X-linked hypophosphatemia: an exploratory analysis of a phase 3 study" (Poster #Sat-025) Lead author: Angela Williams, PhD, Kyowa Kirin International Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT "Individuals with hereditary hypophosphatemia are not prone to early death despite significant earlier development of co-morbidities: a retrospective Danish register study" (Poster #Sun-024) Lead author: Signe Sparre Beck-Nielsen, MD, PhD, Aarhus University Hospital (Denmark) Poster Session II: Sunday, Sept. 29, 2:15 – 3:45 pm EDT "X-linked Hypophosphatemia Community Impact Survey: psychosocial health, symptoms, and self-care" (Poster #SAT-LB 543) Lead author: Jill Simmons, MD, Vanderbilt University Medical Center Late Breaking Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT Bridging evidence gaps to improve clinical decision-making in XLH: "Advancing Patient Evidence in XLH (APEX): rational and design of real-world XLH global data unification program" (Poster #SAT-427) Lead author: Maria Luisa Brandi, MD, PhD, FIRMO Foundation, Florence, Italy and the University Vita-Salute San Raffaele, Milan, Italy Poster Session I: Saturday, Sep 28, 2:15-3:45 pm EDT About X-linked hypophosphatemia X-linked hypophosphatemia is a rare, lifelong, genetic disease that can impact the bones and muscles in both children and adults. In individuals with XLH, the body doesn't hold on to enough phosphorus, which is an essential mineral for bone health. This is due to the production of excess fibroblast growth factor 23 (FGF23), causing the body to release too much phosphorus through the urine. When phosphorus levels are too low (hypophosphatemia), it can cause the softening and weakening of growing bones in children (rickets) and of mature bones in adults (osteomalacia). In children, XLH typically appears as bowed legs or knock knees. Over time, bone weakening can also lead to bone abnormalities in the legs, delayed growth, and short stature. In adults, XLH may cause osteomalacia, fractures and pseudo-fractures, and hypophosphatemia. About CRYSVITA® (burosumab-twza) Injection CRYSVITA is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, which binds to and inhibits the biological activity of FGF23, the underlying cause of hypophosphatemia in XLH. By blocking FGF23, CRYSVITA helps to restore phosphorus reabsorption in the kidneys and increase the production of active vitamin D, which enhances intestinal absorption of phosphate and calcium. U.S. CRYSVITA Indication CRYSVITA is a fibroblast growth factor (FGF23) blocking antibody indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older. Important Safety Information CONTRAINDICATIONS CRYSVITA is contraindicated: In concomitant use with oral phosphate and/or active vitamin D analogs (e.g., calcitriol, paricalcitol, doxercalciferol, calcifediol) due to the risk of hyperphosphatemia. When serum phosphorus is within or above the normal range for age. In patients with severe renal impairment or end stage renal disease because these conditions are associated with abnormal mineral metabolism. WARNINGS AND PRECAUTIONS Hypersensitivity Hypersensitivity reactions (e.g., rash, urticaria) have been reported in patients with CRYSVITA. Discontinue CRYSVITA if serious hypersensitivity reactions occur and initiate appropriate medical treatment. Hyperphosphatemia and Risk of Nephrocalcinosis Increases in serum phosphorus to above the upper limit of normal may be associated with an increased risk of nephrocalcinosis. For patients already taking CRYSVITA, dose interruption and/or dose reduction may be required based on a patient's serum phosphorus levels. Injection Site Reactions Administration of CRYSVITA may result in local injection site reactions. Discontinue CRYSVITA if severe injection site reactions occur and administer appropriate medical treatment. ADVERSE REACTIONS Pediatric Patients Adverse reactions reported in 10% or more of CRYSVITA-treated pediatric XLH patients across three studies are: pyrexia (55%, 44%, and 62%), injection site reaction (52%, 67%, and 23%), cough (52%), vomiting (41%, 48%, and 46%), pain in extremity (38%, 46%, and 23%), headache (34% and 73%), tooth abscess (34%, 15%, and 23%), dental caries (31%), diarrhea (24%), vitamin D decreased (24%, 37%, and 15%), toothache (23% and 15%), constipation (17%), myalgia (17%), rash (14% and 27%), dizziness (15%), and nausea (10%). Postmarketing experience reported in CRYSVITA-treated pediatric XLH patients: blood phosphorus increased. Adult Patients Adverse reactions reported in more than 5% of CRYSVITA-treated adult XLH patients and in at least 2 patients more than placebo in one study are: back pain (15%), headache (13%), tooth infection (13%), restless legs syndrome (12%), vitamin D decreased (12%), dizziness (10%), constipation (9%), muscle spasms (7%), and blood phosphorus increased (6%). Spinal stenosis is prevalent in adults with XLH, and spinal cord compression has been reported. It is unknown if CRYSVITA therapy exacerbates spinal stenosis or spinal cord compression. USE IN SPECIFIC POPULATIONS There are no available data on CRYSVITA use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. Serum phosphorus levels should be monitored throughout pregnancy. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at 1-844-768-3544. There is no information regarding the presence of CRYSVITA in human milk or the effects of CRYSVITA on milk production or the breastfed infant. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for CRYSVITA and any potential adverse effects on the breastfed infant from CRYSVITA or from the underlying maternal condition. PATIENT COUNSELING INFORMATION Advise patients not to use any oral phosphate and/or active vitamin D analog products. Instruct patients to contact their physician if hypersensitivity reactions, injection site reactions, and restless legs syndrome induction or worsening of symptoms occur. You may report side effects to the FDA at (800) FDA-1088 or . You may also report side effects to Kyowa Kirin, Inc. at 1-844-768-3544. For important risk and use information, please see the full Prescribing Information for CRYSVITA. About Kyowa Kirin Kyowa Kirin aims to discover and deliver novel medicines and treatments with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, we have invested in drug discovery and biotechnology innovation for more than 70 years and are currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato oncology, and rare diseases. A shared commitment to our values, to sustainable growth, and to making people smile unites us across the globe. Learn more about Kyowa Kirin at: kyowakirin.com. COR-US-CRY-0049 September 2024 SOURCE Kyowa Kirin WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED