Long-Term CRYSVITA ® ▼ (burosumab) Treatment Reduces the Burden of Disease in Adults With X-Linked Hypophosphataemia (XLH), a Rare Genetic Metabolic Bone Disease
Kyowa Kirin Co., Ltd. (TSE:4151, Kyowa Kirin) today announced the publication of new data highlighting the sustained benefits of treatment with CRYSVITA® (burosumab) in adults with X-linked hypophosphataemia (XLH), a rare genetic metabolic bone disease. The data show that adults with XLH experience substantial pain, stiffness, fatigue and impairment in physical and ambulatory function. Treatment with CRYSVITA was associated with a significant improvement from baseline after 96 weeks.1
The data are from a randomised, double-blind, placebo-controlled, phase 3 study with an open-label extension to assess the efficacy and safety of CRYSVITA in adults with XLH.2 The study met its primary endpoint, showing a statistically significant effect in increasing serum phosphorus concentrations at 24 weeks, compared to placebo.3 After 24 weeks, all patients were switched to CRYSVITA treatment and data was collected on metabolic and biochemical markers, patient reported outcomes (PROs) and measures of mobility up to 96 weeks. This new publication focuses on the results from the PRO analysis and mobility scores.1
At week 96, the study showed statistically significant improvements in PROs, including the Western Ontario and the McMaster Universities Osteoarthritis Index (WOMAC), Brief Pain Inventory–Short Form (BPI-SF) and Brief Fatigue Inventory (BFI), compared to baseline.1 Statistically significant improvements in ambulatory function, measured by the 6-minute walk test (6MWT), were also seen at 96 weeks compared to baseline.1 Data previously published at 48 weeks also showed improvements in some PROs, including stiffness and pain, as well as fracture healing.3
Lead author Pr Karine Briot, Hôpital Cochin, Paris, France said: “The study highlights the many physical challenges faced by adult patients with XLH, including pain, stiffness, fatigue and difficulty walking or physical function. Burosumab treatment has previously been shown to improve phosphate homeostasis in adult XLH patients, compared to placebo. This new analysis suggests that, despite the long-term complications and physical impairment associated with XLH in adults, treatment with burosumab can also improve the physical function and quality of life of adults with XLH over the longer term.”
Tomohiro Sudo, Executive Officer, Head of Global Product Strategy Department of Kyowa Kirin, said: “Kyowa Kirin is committed to improving the lives of people with XLH and their families. One of our areas of focus is to generate new data that improve our understanding of how best to manage and treat XLH. These important new data highlight the many physical challenges that people living with XLH face every day, how their needs could be better met and how Kyowa Kirin is delivering on its purpose, to make people smile.”
The data were published today in the BMJ journal RMD Open, Rheumatic and Musculoskeletal Diseases.1 CRYSVITA is licensed in Europe for the treatment of XLH in children and adolescents aged 1 to 17 years with radiographic evidence of bone disease, and in adults.4
▼This medicinal product is subject to additional monitoring.
About X-linked hypophosphataemia
X-linked hypophosphataemia (XLH) is a rare, genetic disease that causes abnormalities in the bones, muscles, and joints.5,6 XLH is not life-threatening, but its burden is life-long and progressive, and it may reduce a person’s quality of life.7
People with XLH have a genetic defect on the X-chromosome, which causes an excessive loss of phosphate through the urine and poor absorption from the gut due to excess of a hormone known as fibroblast growth factor-23 (FGF23), resulting in chronically low levels of phosphate in the blood.4,8 Phosphate is a key mineral needed for maintaining the body’s energy levels, muscle function, and the formation of healthy bones and teeth.9,10 While there is no cure for XLH, therapies aimed at helping to restore and maintain phosphate to normal levels within the body may help to improve the progression of disease symptoms.2
XLH is the most common form of hereditary rickets.11 It can sometimes appear in individuals with no family history of the disease but is usually passed down from a parent who carries the defective gene.12
About CRYSVITA® (burosumab)
CRYSVITA (burosumab) was created and developed by Kyowa Kirin and is a recombinant fully human monoclonal IgG1 antibody against the phosphaturic hormone fibroblast growth factor 23 (FGF23). FGF23 is a hormone that reduces serum levels of phosphate by regulating phosphate excretion and active vitamin D production by the kidney. Phosphate wasting and resulting hypophosphataemia in X-linked hypophosphataemia (XLH) is caused by excess FGF23. CRYSVITA is designed to bind to, and thereby inhibit, the biological activity of FGF23. By blocking excess FGF23 in patients, CRYSVITA is intended to increase phosphate reabsorption from the kidney and increase the production of active vitamin D, which enhances intestinal absorption of phosphate and calcium.
CRYSVITA has been available for clinical use since 2018. The first approval came from the European Commission, that granted a conditional marketing authorisation for CRYSVITA for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older and adolescents with growing skeletons. In 2020, this authorisation was subsequently expanded to include older adolescents and adults.2
CRYSVITA is approved by the US Food and Drug Administration (FDA) for patients with XLH aged 6 months and older and by Health Canada for patients with XLH aged one year and older.13,14
In 2019, CRYSVITA received approval from Japan's Ministry of Health, Labour and Welfare for the treatment of FGF23-related hypophosphataemic rickets and osteomalacia. In 2020, CRYSVITA was reimbursed by National Health Insurance (NHI) in Japan as a self-injection presentation for the treatment of FGF23-related hypophosphataemic rickets and osteomalacia.
In January 2020, Swissmedic approved CRYSVITA for the treatment of adults, adolescents and children (one year of age and older) with XLH.15
In June 2020, the U.S. Food and Drug Administration (FDA) approved CRYSVITA for patients aged two and older with tumour-induced osteomalacia (TIO), a rare disease that is characterised by the development of tumours that cause weakened and softened bones.16
Kyowa Kirin and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE: Ultragenyx) have been collaborating in the development and commercialisation of CRYSVITA globally, based on the collaboration and licence agreement between Kyowa Kirin and Ultragenyx.
About Kyowa Kirin
Kyowa Kirin strives to create and deliver novel medicines with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company with a more than 70-year heritage, the company applies cutting-edge science including an expertise in antibody research and engineering, to address the needs of patients and society across multiple therapeutic areas including Nephrology, Oncology, Immunology/Allergy and Neurology. Across our four regions – Japan, Asia Pacific, North America and EMEA/International – we focus on our purpose, to make people smile, and are united by our shared values of commitment to life, teamwork/Wa, innovation, and integrity. You can learn more about the business of Kyowa Kirin at: https://www.kyowakirin.com/
Kyowa Kirin International
Galabank Business Park
Galashiels, TD1 1QH
1 Briot K, Portale AA, Brandi ML, et al. RMD Open 2021;7:e001714. doi: 10.1136/rmdopen-2021-001714.
2 Insogna KL, Rauch F, Kamenický P, et al. Burosumab improved histomorphometric measures of osteomalacia in adults with X-linked hypophosphatemia: a Phase 3, single-arm, international trial. J Bone Miner Res. 2019;34:2183-2191.
3 Portale AA, Carpenter TO, Brandi ML, et al. Calcif Tissue Int 2019;105:271–84.
4 European Medicines Agency. CRYSVITA EPAR product information. Summary of Product Characteristics. Available at: Crysvita, INN-burosumab; (europa.eu). Last updated: June 2021. Last accessed: July 2021.
5 Linglart A, Biosse-Duplan M, Briot K, et al. Therapeutic management of hypophosphatemic rickets from infancy to adulthood. Endocr Connect. 2014;3:R13-30.
6 Haffner D, Emma F, Eastwood DM, et al. Consensus Statement. Evidence-based guideline. Clinical practice recommendations for the diagnosis and management of X-linked hypophosphatemia. Nat Rev Nephrol. 2019;15;435-455.
7 Skrinar A, Dvorak-Ewell M, Evins A, et al. The lifelong impact of X-linked hypophosphatemia: Results from a burden of disease survey. J Endocr Soc. 2019;3:1321-1334.
8 Beck-Nielsen SS, Mughal Z, Haffner D, et al. FGF23 and its role in X-linked hypophosphatemia-related morbidity. Orphanet J Rare Dis. 2019;14:58.
9 Pesta D, Tsirigotis DN, Befroy DE, et al. Hypophosphatemia promotes lower rates of muscle ATP synthesis. The FAESB Journal. 2016;39:3378-3387.
10 Unnanuntana A, Rebolledo BJ, Khair MM, et al. Diseases affecting bone quality: beyond osteoporosis. Clin Orthop Relat Res. 2011;469:2194-2206.
11 Carpenter TO, Imel EA, Holm IA, et al. A clinician's guide to X-linked hypophosphatemia. J Bone Miner Res. 2011;26:1381-8.
12 National Center for Advancing Translational Sciences. X-linked hypophosphatemia. Available at: https://rarediseases.info.nih.gov/diseases/12943/x-linked-hypophosphatemia. Last updated: February 2018. Last accessed: July 2021.
13 Health Canada. Regulatory Decision Summary – CRYSVITA. Available at: https://hpr-rps.hres.ca/reg-content/regulatory-decision-summary-detail.php?linkID=RDS00463. Last updated: April 2020. Last accessed: April 2021.
14 Available at : https://www.kyowakirin.com/media_center/news_releases/2019/e20190930_01.html. Last accessed: July 2021
15 Swissmedic. Crysvita, injektionslösung (burosumabum). Available at: https://www.swissmedic.ch/swissmedic/en/home/humanarzneimittel/authorisations/new-medicines/vrysvita-injektionsloesung_burosumabum.html. Last updated: January 2020. Last accessed: July 2021.
16 FDA. Available at: FDA Approves First Therapy for Rare Disease that Causes Low Phosphate Blood Levels, Bone Softening | FDA. Last accessed: July 2021
To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.
Kyowa Kirin Co., Ltd.:
Kyowa Kirin International:
+44 (0) 7769 656073
About Business Wire
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
The Spotlight on Iceland’s Capital City Shines Even Brighter With the Arrival of The Reykjavik EDITION21.10.2021 17:30:00 CEST | Press release
A flourishing culinary hotspot with cool cafés, a rollicking nightlife and an epic music scene, the spotlight is shining brightly on Iceland’s hip capital city and, with typical finesse, the arrival of The Reykjavik EDITION further cements EDITION Hotels' uncanny ability to land in just the right place at the right time. “Reykjavik is a really cool, young city -perfect for our brand,” says Ian Schrager, the visionary pioneer of the boutique hotel concept, PUBLIC and EDITION creator. “We think this is Reykjavik’s time and we’re right here at the very heart of it and at the perfect time.” This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211021005764/en/ The Reykjavik EDITION (Photo: Business Wire) Opening in preview on November 9th, 2021, The Reykjavik EDITION will set a new standard as the city’s first truly luxury hotel experience, combining the best of the Icelandic capital with the personal, intimate and individual experienc
MediaTek Selected to Power Vewd for Automotive Platform21.10.2021 16:41:00 CEST | Press release
Vewd, the leading provider of OTT software solutions, today announced that MediaTek’s Autus I20 (MT2712) chipset was selected as the first reference platform to power Vewd for Automotive, a white-labeled, cloud-managed content aggregation and monetization solution for in-vehicle infotainment equipment providers and car manufacturers. MediaTek’s customer base, streaming ecosystem partnerships, and strong technical capabilities make it the strongest supplier to meet the needs of car manufacturers. Together, Vewd and MediaTek will target Strategy Analytics’ estimated 70 million connected cars on the road by 2025. Vewd for Automotive solves key rear-seat content challenges facing manufacturers through a turnkey solution ready for deployment. For manufacturers, content in the car represents an increasing consumer demand, along with the demand for connectivity that provides additional after-sale branding and revenue opportunities. By partnering with content providers, manufacturers can enabl
Biognosys to Present Major Scientific and Technological Advances at the ASMS 2021 Mass Spectrometry Conference21.10.2021 16:00:00 CEST | Press release
Biognosys, a leading inventor and developer of mass spectrometry-based proteomics solutions, today announced they will be presenting major scientific and technological advances on their proprietary proteomics research services, software, and kits at the American Society for Mass Spectrometry (ASMS) Annual Conference from October 31st to November 4th in Philadelphia (USA). Biognosys will present a record number of 3 oral presentations, 10 scientific posters, 2 poster collaborations, 1 workshop panel, and 2 Spectronaut™ breakfast seminars. In addition, their team of scientific experts will be present at booth #224 to answer questions and demo software. Further demo sessions will be offered at the Bruker Daltonics booth #719. Collectively, this presence demonstrates Biognosys’ significant contributions to transforming life science and clinical research with next-generation proteomics, particularly in the areas of plasma proteomics, immunopeptidomics, and proteomics data analysis. Lukas Re
SHV Energy Implements Global Remote LPG Tank Monitoring With Sigfox Netherlands’ 0G Network and Reduces Carbon Emissions21.10.2021 16:00:00 CEST | Press release
SHV Energy and Sigfox are rolling out a global LPG tank level monitoring solution. The implementation has started in France, Belgium and Germany and is already generating lower carbon emissions and improved customer satisfaction. Over the next three years, 50,000 units will be rolled out worldwide. Sigfox’s IoT (Internet of Things) asset monitoring capability enables SHV Energy to reduce the cost of tank level monitoring, while increasing efficiency and productivity. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211021005065/en/ SHV Energy LPG tank (Photo: Sigfox) SHV Energy, a leading global distributor of off-grid energy such as LPG, LNG, biofuels and renewables, with 30 million customers in 25 countries and 16,700 employees, selected to work with Sigfox, a world’s leading IoT communication service provider and 0G network pioneer. SHV Energy set-up a Telemetry Centre of Excellence and used Sigfox’ solutions and partner At
SSZN Selects Newsight's NSI1000 Chip for Its Advanced Industry 4.0 Production Line Sensors21.10.2021 15:50:00 CEST | Press release
Newsight Imaging Ltd., an innovative semiconductor company developing machine vision sensors, spectral vision chips and systems, announces a commercial agreement with SSZN. SSZN is a leader in developing smart industry 4.0 solutions for supporting automated production line supervision. Newsight will provide SSZN with the NSI1000 - its new and advanced sensor chip. The deal follows a successful design-win where SSZN conducted an evaluation using the NSI1000 chip, closely supported by Newsight's engineering teams in Israel and Shenzhen. The first order of 100,000 chips will be supplied in the upcoming months. The NSI1000 CMOS Image sensor chip, launched by Newsight last year, consists of 32 lines of 1024 pixels and it supports multiple modes, such as multi-triangulation for state-of-the-art accuracy and fast operation on production line inspection. The sensor implements Newsight's enhanced Time of Flight (eTOF™) technology for advanced, long-range, and high-resolution 3D depth image capt
Esper and Lenovo Collaborate on Android Device Deployment in Enterprises21.10.2021 15:05:00 CEST | Press release
DevOps for Devices leader Esper and Lenovo™are teaming up to place Esper software on key models of Lenovo Android tablets and other intelligent business devices. This collaboration allows enterprises that manage large fleets of devices to focus on their critical line-of-business applications, leaving the device infrastructure and management tasks to Lenovo and Esper’s joint solution. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211021005372/en/ Lenovo K10 Tablet (Photo: Business Wire) As the fastest-growing tablet maker (YoY) last quarter1, Lenovo’s expertise in smarter device design, engineering and user experiences combines with Esper’s modern, DevOps-powered device infrastructure software that lets organizations of every size and segment (e.g. healthcare, retail, hospitality), easily set up, manage, better secure, and maintain large fleets of devices on their journey to digital transformation. “With proprietary features
Finalist Teams Selected in $10M ANA Avatar XPRIZE Competition21.10.2021 15:00:00 CEST | Press release
XPRIZE, the world’s leader in designing and operating incentive competitions to solve humanity’s grand challenges, announced today that 15 teams from 8 countries are advancing to the finals round of the $10M ANA Avatar XPRIZE competition. Sponsored by All Nippon Airways (ANA), Japan’s largest airline, the Avatar XPRIZE is a four-year global competition focused on the development of an avatar system that will deploy a human’s senses, actions, and presence to a remote location in real time, leading to a more connected world. In the future, avatars could help provide critical care and deploy immediate responses in emergency situations, or offer opportunities for exploration and new ways of collaboration, stretching the boundaries of what is possible and maximizing the impact of skill and knowledge sharing. These avatars must demonstrate the ability to execute tasks across a variety of real-world scenarios and convey a sense of presence for both the operator and the recipient in those inte
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.Visit our pressroom