Kyowa Kirin Announces European Commission (EC) Approval of CRYSVITA® (burosumab) for the Treatment of X-Linked Hypophosphataemia (XLH) in Older Adolescents and Adults
Kyowa Kirin Co., Ltd. (TSE: 4151, Kyowa Kirin) today announced that the European Commission (EC) has approved CRYSVITA® (burosumab) for use in older adolescents and adults with the rare disease X-linked hypophosphataemia (XLH). CRYSVITA was previously approved for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older and adolescents with growing skeletons. With this expanded approval, all adolescents with radiographic evidence of bone disease, regardless of growth status, as well as adults with XLH are now also eligible for treatment with CRYSVITA.4
XLH is a life-long and progressive disease that typically presents in early childhood, causing lower limb deformities, stunted growth, and bone and joint pain.5 Symptoms such as dental abscesses, osteoarthritis, enthesopathy (issues with the tendons), and hearing loss may also develop during adulthood.6,7,8 As a result of the disease, some adults may require special equipment to improve their mobility.9,10 The physical limitations as well as pain and stiffness caused by XLH can affect people’s ability to work and socialise, their emotional wellbeing, and their capacity for self-care.9
“Until now, adults living with XLH have had limited treatment options for this progressive, disabling condition,” said Dr Karine Briot, Hôpital Cochin, Paris, France. “Today’s approval is an important advance as it means that for the first time these adult XLH patients have a treatment option developed to target the underlying processes that cause this challenging disease.”
“Today’s decision from the European Commission is a significant milestone for the management of XLH, a progressive and life-long disease that profoundly impacts the lives of both children and adults,” said Abdul Mullick, President of Kyowa Kirin International. “As part of our commitment to life, we strive to answer the unmet medical needs of patients, their families, and the medical professionals who care for them. With this approval, older adolescents and adults with XLH will also be able to benefit from treatment with CRYSVITA, the only therapy that targets the underlying pathophysiology of this disease. We now will focus on ensuring access for as many people as possible in this expanded group of eligible patients.”
The application to expand the marketing authorisation was based on data from two Phase 3 studies: the Phase 3 UX023-CL303 study, a randomised, double-blind, placebo-controlled trial investigating the safety and efficacy of burosumab in adults with XLH, and the Phase 3 UX023-CL304 study, an open-label, single-arm study investigating the effects of burosumab on osteomalacia (softening of the bones) in adults with XLH. These two studies found that burosumab increased and maintained serum phosphate levels in the normal range, helped to heal pseudofractures and fractures related to osteomalacia, and improved osteomalacia. Other endpoints showed that patients had less pain and stiffness, and their physical functioning and mobility improved with time.11,12,13 The safety profile of burosumab was consistent with that observed in other burosumab studies, with adverse events including injection site reactions, hyperphosphataemia and hypersensitivity. There were no treatment-related serious adverse events.11,12
The Kyowa Kirin Group companies strive to contribute to the health and well-being of people around the world by creating new value through the pursuit of advances in life sciences and technologies.
About X-linked hypophosphataemia
X-linked hypophosphataemia (XLH) is a rare, genetic disease that causes abnormalities in the bones, muscles, and joints.1,2,3 XLH is not life-threatening, but its burden is life-long and progressive, and it may reduce a person’s quality of life.5
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, resulting in chronically low levels of phosphate in the blood.5,14 Phosphate is a key mineral needed for maintaining the body’s energy levels, muscle function, and the formation of healthy bones and teeth.15,16 While there is no cure for XLH, therapies aimed at helping to restore phosphate to normal levels within the body may help to improve the symptoms of the disease.12
XLH is the most common form of hereditary rickets.17 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.18
About CRYSVITA® (burosumab)
CRYSVITA (burosumab) was created 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 excessive levels and activity of 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 vitamin D, which enhances intestinal absorption of phosphate and calcium.
In 2018, the European Commission 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.4 In the same year, CRYSVITA received approval from the US Food and Drug Administration (FDA) and Health Canada for paediatric and adult use.19,20
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 January 2020, Swissmedic approved CRYSVITA for the treatment of adults, adolescents and children (one year of age and older) with XLH.21
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 commits to innovative drug discovery driven by state-of-the-art technologies. The company focuses on creating new values in the four therapeutic areas: nephrology, oncology, immunology/allergy and neurology. Under the Kyowa Kirin brand, the employees from 40 group companies across North America, EMEA, and Asia/Oceania unite to champion the interests of patients and their caregivers in discovering solutions wherever there are unmet medical needs.
You can learn more about the business of Kyowa Kirin at: https://www.kyowakirin.com/
1 Linglart A, Biosse-Duplan M, Briot K, et al. Therapeutic management of hypophosphatemic rickets from infancy to adulthood. Endocr Connect. 2014;3:R13-30.
2 Orphanet. X-linked hypophosphatemia. Available at: https://www.orpha.net/consor/cgi-bin/OC_Exp.php?Expert=89936. Last updated: January 2012. Last accessed: September 2020.
3 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.
4 European Medicines Agency. CRYSVITA EPAR product information. Summary of Product Characteristics. Available at: https://www.ema.europa.eu/en/documents/product-information/crysvita-epar-product-information_en.pdf. Last updated: March 2020. Last accessed: September 2020.
5 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.
6 Lee JY, Imel EA. The changing face of hypophosphatemic disorders in the FGF-23 era. Pediatr Endocrinol Rev. 2013;10:367-379.
7 Che H, Roux C, Etcheto A, et al. Impaired quality of life in adults with X-linked hypophosphatemia and skeletal symptoms. Eur J Endocrinol. 2016;174:325-333.
8 Ruppe MD. X-Linked Hypophosphatemia. In: Adam MP, Ardinger HH, Pagon RA, et al., editors. GeneReviews®. Seattle (WA): University of Washington, 1993-2018. Available at: https://www.ncbi.nlm.nih.gov/books/NBK83985/. Last accessed: September 2020.
9 Lo SH, Lachmann R, Williams A, et al. Exploring the burden of X-linked hypophosphatemia: a European multi-country qualitative study. Qual Life Res. 2020;29:1883-1893.
10 Theodore-Oklota C, Bonner N, Spencer H, et al. Qualitative research to explore the patient experience of X-linked hypophosphatemia and evaluate the suitability of the BPI-SF and WOMAC® as clinical trial end points. Value Health. 2018;21:973-983.
11 Insogna KL, Briot K, Imel EA, et al. A randomized, double-blind, placebo-controlled, Phase 3 trial evaluating the efficacy of burosumab, an anti-FGF23 antibody, in adults with X-linked hypophosphatemia: week 24 primary analysis. J Bone Miner Res. 2018;33:1383-1393.
12 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.
13 Portale AA, Carpenter TO, Brandi ML, et al. Continued beneficial effects of burosumab in adults with X-linked hypophosphatemia: Results from a 24-week treatment continuation period after a 24-week double-blind placebo-controlled period. Calcif Tissue Int. 2019;105:271-284.
14 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.
15 Pesta D, Tsirigotis DN, Befroy DE, et al. Hypophosphatemia promotes lower rates of muscle ATP synthesis. The FAESB Journal. 2016;39:3378-3387.
16 Unnanuntana A, Rebolledo BJ, Khair MM, et al. Diseases affecting bone quality: beyond osteoporosis. Clin Orthop Relat Res. 2011;469:2194-2206.
17 Carpenter TO, Imel EA, Holm IA, et al. A clinician's guide to X-linked hypophosphatemia. J Bone Miner Res. 2011;26:1381-8.
18 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: September 2020.
19 CRYSVITA Prescribing Information. Available at: https://www.ultragenyx.com/file.cfm/29/docs/Crysvita_Full_Prescribing_Information.pdf. Last updated: June 2020. Last accessed: September 2020.
20 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: September 2020.
21 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: September 2020.
Contacts for Kyowa Kirin Co., Ltd.:
Contacts for Kyowa Kirin International:
+44 (0) 7769 656073
About Business Wire
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
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
PMI Announces Medicago to Supply Up to 76 Million Doses of Its Plant-Derived COVID-19 Vaccine Candidate23.10.2020 21:00:00 EEST | Press release
Since 2008, Philip Morris Investments B.V. (PMIBV), a subsidiary of Philip Morris International (PMI) (NYSE: PM), has been a shareholder of Medicago (in which it currently holds an approximately one-third equity stake) and has supported Medicago’s innovative plant-derived research and development focused on vaccines. The investment is consistent with PMI’s own efforts to leverage science and innovation. Japan-based Mitsubishi Tanabe Pharma Corporation (MTPC) is the majority shareholder and PMIBV’s partner in Medicago. Among other things, PMIBV and MTPC will contribute additional funding to support Medicago’s efforts to develop a COVID-19 vaccine candidate. Medicago, a biopharmaceutical company headquartered in Quebec City, announced that it reached an agreement with Public Services and Procurement Canada (PSPC) to supply up to 76 million doses of its vaccine candidate for COVID-19, subject to Health Canada approval. Innovation, Science & Economic Development (ISED), another department
Fujikura Enters 5G mmWave Infrastructure Market With the Introduction of Industry’s Highest Performance, and Low Power Consumption Phased Array Antenna Module (PAAM)23.10.2020 11:00:00 EEST | Press release
Fujikura Ltd. (TOKYO:5803) (President & CEO: Masahiko Ito) today announced the introduction of industry’s most advanced and highly integrated Phased Array Antenna Module (PAAM) for 5G mmWave operating in 3GPP bands n257 (28 GHz), n258 (26 GHz) and n261 (27 GHz). The Fujikura PAAM is the industry’s highest performance PAAM targeting indoor and outdoor applications such as 5G mmWave Fixed Wireless Access (FWA), 5G mmWave Mobile Wireless Access (MWA), 5G mmWave wireless backhaul, and other emerging applications. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201023005099/en/ Fig.1 Block diagram of Fujikura PAAM. (Graphic: Business Wire) Key Differentiating Features of the Fujikura PAAM The Fujikura PAAM is a high performance phased array antenna module with a novel integrated antenna, and supports concurrent dual polarized beams in both transmission and reception (Fig.1). The PAAM integrates best-in-class mmWave-ICs developed i
VAAST Bikes Now Available in Europe23.10.2020 10:02:00 EEST | Press release
VAAST Bikes, a forward-thinking bicycle manufacturer known for its innovative Allite® Super Magnesium™ frames, has signed an agreement with German cycling-specialist retailer giant Internetstores (Fahrrad.de/Brügelmann.de/Bikester). Two of VAAST’s key bicycle models – including the gravel-ready all-road A/1 bike, and the urban U/1 – are now stocked at Internetstores’ eCommerce sites. Both VAAST models will be available to order online for delivery in fourteen locations across Europe via Fahrrad.de/Brügelmann.de in Germany and the partner site Bikester in 13 countries, including the UK, France, the Netherlands, Belgium, Switzerland, Austria, Poland, Denmark, Italy, Sweden, Norway, Finland and Spain. Customers in Germany can benefit from personal service in Fahrrad.de’s five stores and 190 local service partners, while eCommerce customers Europe-wide will be able to receive their new VAAST bicycle within just days of ordering online. VAAST’s unique position as the only bicycle manufactur
Mitsubishi Chemical Holdings Corporation: Notice Regarding Change in Representative Corporate Executive Officers23.10.2020 09:40:00 EEST | Press release
Mitsubishi Chemical Holdings Corporation (“MCHC”) (TOKYO: 4188) announced that, at the meeting of its Board of Directors held today, it resolved change of representative corporate executive officers as outlined below. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201022006285/en/ Jean-Marc Gilson (Photo: Business Wire) 1. Change in Representative Corporate Executive Officers (Appointed) Name: Jean-Marc Gilson Title: Representative Corporate Executive Officer President and Chief Executive Officer (Retiring) Name: Hitoshi Ochi Title: Representative Corporate Executive Officer President and Chief Executive Officer 2. Biographical Sketch of New Representative Corporate Executive Officer Refer to the appendix 3. Scheduled Date of Appointment April 1, 2021 4. Reason for the Change MCHC converted to a company with a nominating committee, etc. in June 2015. Since then, the Nominating Committee has carried out preparations to proper
MYAH Wins Prestigious Silmo D’Or Award23.10.2020 08:00:00 EEST | Press release
Topcon Healthcare, a leading provider of medical devices and software solutions for the global eye care community, announced today that its MYAH myopia and dry eye management device has won the 2020 Silmo D’Or Award in the Material/Equipment category. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201022005242/en/ Topcon Healthcare announced today that its MYAH myopia and dry eye management device has won the 2020 Silmo D’Or Award. (Photo: Business Wire) The Silmo D’Or Awards are presented each year to recognize creativity in optical product design, innovation and technology. This year marked the 27th annual awards ceremony, which was held virtually and live streamed online on Saturday, October 3rd. The award committee was proud to recognize forward-thinking companies who have continued to launch products and propel the industry forward despite the global pandemic. MYAH came out on top in the Material/Equipment category and
ABB: Q3 2020 Results23.10.2020 07:46:00 EEST | Press release
“Third quarter revenues in all business areas were still dampened due to the impact of COVID-19, although a strong recovery in China and ongoing cost mitigation efforts supported a strong underlying performance. On the upside, the integration of GEIS and turnaround of Installation Products in Electrification is starting to bear fruit and Motion is performing robustly. Robotics and Industrial Automation, on the other hand, are taking more time to recover,” said Björn Rosengren, CEO of ABB. “We are pushing ahead with the decentralization of the group and the ongoing review of our portfolio, while carrying out our share buyback program as planned. We look forward to presenting further details on our strategic progress at our Capital Markets Day on November 19.” KEY FIGURES CHANGE CHANGE ($ millions, unless otherwise indicated) Q3 2020 Q3 2019 US$ Comparable 9M 2020 9M 2019 US$ Comparable Orders 6,109 6,688 -9% -8% 19,509 21,702 -10% -7% Revenues 6,582 6,892 -4% -4% 18,952 20,910 -9% -7% I
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