Business Wire

Kyowa Kirin Announces EU Approval for the Self-administration of CRYSVITA ® ▼ (burosumab) to Treat X-Linked Hypophosphataemia (XLH)

Share

Kyowa Kirin Co., Ltd. (TSE:4151, Kyowa Kirin) today announced that CRYSVITA® (burosumab) has been approved for the option of self-administration in the European Union (EU) for the treatment of X-linked hypophosphataemia (XLH), a rare metabolic bone disease that impacts children and adults. The approval means that some patients or carers may be suitable to administer CRYSVITA themselves, at the recommendation of the treating physician, in its licensed indication for the treatment of XLH in children and adolescents aged 1 to 17 years with radiographic evidence of bone disease, and in adults.1

Dr Raja Padidela, Consultant Paediatric Endocrinologist at Royal Manchester Children’s Hospital, UK said: “XLH is a progressive, disabling condition, causing rickets, lower limb deformities, stunted growth, bone and joint pain. Now CRYSVITA is approved in Europe for self-administration, healthcare professionals will have the option to help XLH patients and their carers gain more independence. Adults with XLH and their carers, or the parents or guardians of young children or adolescents with XLH, may benefit from CRYSVITA’s self-administration option. The self-administration of CRYSVITA is at the discretion of a treating physician, providing those self-administering are competent and comfortable doing so.”

Abdul Mullick, President of Kyowa Kirin International, said: “This approval is another significant milestone for the treatment of people living with XLH. Kyowa Kirin is committed to improving the lives of patients and their families by giving healthcare professionals better ways to care for them. By gaining EU approval for the self-administration of CRYSVITA, we have created another valuable option in the care of children, adolescents and adults with XLH. The self-administration of CRYSVITA is a great example of how Kyowa Kirin is meeting physician and patient needs and delivering on our purpose, to make people smile.”

CRYSVITA is administered by subcutaneous injection.2 CRYSVITA treatment will still need to be initiated by a physician experienced in the management of patients with metabolic bone diseases. Subsequently, if the patient is receiving a stable dose, the physician may recommend that administration can be performed by the patient or carer following appropriate training. The first self-administered dose after drug initiation or dose change will need to be conducted under the supervision of a healthcare professional.

▼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.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.4

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.4,5 Phosphate is a key mineral needed for maintaining the body’s energy levels, muscle function, and the formation of healthy bones and teeth.6,7 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.8

XLH is the most common form of hereditary rickets.9 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.10

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.11,12

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.13

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.14

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
http://www.international.kyowa-kirin.com / www.kyowakirin.com
Galabank Business Park
Galashiels, TD1 1QH
United Kingdom

References


1 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.

2 Linglart A, Biosse-Duplan M, Briot K, et al. Therapeutic management of hypophosphatemic rickets from infancy to adulthood. Endocr Connect. 2014;3:R13-30.

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 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.

5 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.

6 Pesta D, Tsirigotis DN, Befroy DE, et al. Hypophosphatemia promotes lower rates of muscle ATP synthesis. The FAESB Journal. 2016;39:3378-3387.

7 Unnanuntana A, Rebolledo BJ, Khair MM, et al. Diseases affecting bone quality: beyond osteoporosis. Clin Orthop Relat Res. 2011;469:2194-2206.

8 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.

9 Carpenter TO, Imel EA, Holm IA, et al. A clinician's guide to X-linked hypophosphatemia. J Bone Miner Res. 2011;26:1381-8.

10 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.

11 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.

12 Available at : https://www.kyowakirin.com/media_center/news_releases/2019/e20190930_01.html. Last accessed: July 2021

13 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.

14 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.

Contact information

Contacts for Kyowa Kirin Co., Ltd.:
Media

Hiroki Nakamura
+81-3-5205-7205
Email: media@kyowakirin.com

Contacts for Kyowa Kirin International:
Media
Stacey Minton
+44 (0) 7769 656073
Email: Stacey.Minton@kyowakirin.com

About Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

(c) 2018 Business Wire, Inc., All rights reserved.

Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.

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

Ipsen Strengthens Its Pre-clinical Oncology Pipeline With an Exclusive Worldwide-collaboration With BAKX Therapeutics Inc. for BKX-001, Targeting the Apoptosis Pathway27.7.2021 19:01:00 CEST | Press release

Regulatory News: Disclaimer: Intended for international media and investor audiences only Ipsen (Euronext: IPN; ADR: IPSEY) and BAKX Therapeutics Inc. have signed an exclusive worldwide-collaboration agreement to research, develop, manufacture and commercialize BKX-001 as a potential treatment for leukemia, lymphoma and solid tumors. Apoptosis is the naturally occurring process of programmed cell death. Deregulated apoptosis can lead to uncontrolled cell division and the development of a tumor.2 The apoptosis cell-signaling pathway has been proven to be a target for cancer therapy with the development and approval of BCL-2 inhibitors for the treatment of certain hematological malignancies.3 BAX is a novel target in this pathway that is downstream of all anti-apoptotic proteins like BCL-2, BCL-XL, MCL-1 etc.4 Direct activation of BAX has several possible outcomes as an investigational cancer therapy, potentially addressing multiple tumor types and the resistance encountered while target

Daikin Industries Becomes OCSiAl Shareholder27.7.2021 18:55:00 CEST | Press release

Japanese giant Daikin Industries, Ltd, has become a shareholder of the world’s largest producer of graphene nanotubes, OCSiAl, following three years of collaboration in developing graphene nanotube applications as a next-generation additive. Together, the companies are determined to develop high-performance materials and establish new markets. The signing of a share subscription agreement between Daikin Industries, a world-leading air conditioning company and a pioneer in fluorine technologies, and OCSiAl, the world’s largest producer of graphene nanotubes and related industrial solutions, was supported by prior joint developments and market evaluation. In accordance with the terms of the agreement, the valuation of OCSiAl is about USD $2 billion. Along with the subscription agreement, the two companies have signed a cooperation agreement to develop, produce, and market graphene nanotube solutions for next-generation Li-ion batteries and fluoropolymers, which comprise a substantial par

V.E, part of Moody’s ESG Solutions, provides Second Party Opinion on Colombia’s Sovereign Green Bond Framework and Inaugural Issuance27.7.2021 17:58:00 CEST | Press release

V.E, part of Moody’s ESG Solutions, published today a Second Party Opinion (SPO) on Colombia’s Sovereign Green Bond Framework and inaugural issuance. The SPO assesses four main components: the framework’s level of contribution to sustainability, its alignment with international standards, its coherence with Colombia’s strategic sustainability priorities, and Colombia’s environmental, social and governance (ESG) performance. “Proceeds from the green instruments issued under Colombia’s framework will provide a ‘robust’ contribution to sustainability, the second-highest level on our four-point scale,” said Adriana Cruz Felix, Head of Sustainable Finance Research at V.E. “The issuance will fund projects to protect biodiversity as well as build climate change resilience and adaptation, thereby reinforcing the capacity of Colombia’s institutions to address key environmental challenges.” In V.E’s opinion, the framework and portfolio of eligible expenditures are aligned with the four core comp

DYSIS Announces New Compact and Portable Colposcope Design with Computer-Aided Cervical Mapping27.7.2021 17:30:00 CEST | Press release

DYSIS Medical Inc. is excited to announce the launch of DYSIS® View, a compact and portable colposcope, which includes the company’s innovative computer-aided cervical mapping technology that helps healthcare professionals detect cervical lesions more clearly. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210727005846/en/ DYSIS View | Computer-Aided Colposcopy with Cervical Mapping (Photo: Business Wire) The DYSIS family of colposcopes include the novel DYSISmap, which is a color-coded summary of the acetowhitening effect of the patient’s cervix. Colors are allocated on the map depending on the acetowhitening changes measured by DYSIS during the examination. This data helps healthcare professionals improve biopsy selection. In the IMPROVE-COLPO study, when map-assisted biopsies were added to colposcopy exams, detection of patients with CIN2+ increased by 44%1. “We are thrilled to bring DYSIS View to market to complement our

Exchanger Industries Limited Acquires HRS Heat Exchangers27.7.2021 17:00:00 CEST | Press release

Exchanger Industries Limited (“EIL”), the Canadian market leader and globally recognized designer and manufacturer of heat transfer products for the energy, petrochemical, industrial and clean power generation sectors, has acquired HRS Heat Exchangers Ltd. ("HRS"). Financial terms were not disclosed. HRS is widely recognized as the pre-eminent, international specialist supplier of heat exchangers and custom process systems across the environmental, food, beverage, and pharmaceutical sectors. The seasoned HRS management team will be retained by EIL and play a critical role in executing plans for robust international expansion afforded by the strategic combination of two distinct, yet complementary industry leaders. “For decades, we’ve worked hard on behalf of our customers in Canada, the U.S. and 25 countries worldwide, consistently providing them with mission-critical, innovative solutions in some of the most demanding environments,” stated Mark el Baroudi, EIL’s CEO. “The HRS acquisit

Quanergy’s 3D LiDAR Helps Waste Incineration Plant Optimize Recycling Process and Reduce Environmental Impact in China27.7.2021 16:18:00 CEST | Press release

Quanergy Systems, Inc., a leading provider of OPA-based solid state LiDAR sensors and smart 3D solutions for automotive and IoT, today announced a new smart LiDAR deployment automating waste plant operations, to ensure process efficiency and minimize the environmental impact of a waste-to-energy incineration plant in China. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210727005789/en/ Quanergy’s 3D LiDAR Helps Waste Incineration Plant Optimize Recycling Process and Reduce Environmental Impact in China (Graphic: Business Wire) Deployed in collaboration with Hunan University, one of China’s top engineering research universities, and Hunan Qiaokang Bridge Health Intelligent Technology for Puxiang Bioenergy, Quanergy’s high accuracy M-Series 3D LiDAR sensors were applied to scan the surface of the waste piles at the incineration plant and create a 3D point cloud image, to accurately calculate the volume of the waste. The LiDAR

Andersen Global Enhances Gambian Foothold with Payce Consulting27.7.2021 15:30:00 CEST | Press release

Andersen Global continues its Africa expansion with the addition of collaborating firm Payce Consulting, adding dimension to the organization’s existing presence in The Gambia. Payce Consulting, founded in 2014 by Partner Paul Gaye, serves the public and private sectors at the local and international level including financial institutions, education, industrial and commerce, transportation, fish processing and export, hotel and leisure, seaport, energy, agriculture, information technology and government agencies. The tax and advisory firm provides corporate finance, business advisory, business structuring and insolvency, outsourcing, financial services and portfolio management services to clients. “Our client-focused approach, extensive industry knowledge and commitment to best-in-class service have allowed us to develop and maintain strong client relationships,” Paul said. “Our collaboration with Andersen Global is the next step for our firm as we look to provide our clients with the