SUCH –

Kyowa Kirin Co. Ltd. (TSE: 4151, Kyowa Kirin) announced today that CRYSVITA® (Burosumab) has received EU approval for the treatment of X-linked hypophosphatemia (XLH), a rare metabolic bone disease that can affect children and adults, for self-administration. Approval means that certain patients or caregivers may be suitable for self-administering CRYSVITA on the recommendation of the treating physician for the approved indication for the treatment of XLH in children and adolescents aged 1 to 17 years with radiographic evidence of a bone disease as well as in adults.1

Dr. Commented Raja Padidela, Consultant Pediatric Endocrinologist at Royal Manchester Children’s Hospital, UK: “XLH is a progressive disability causing rickets, lower limb deformity, stunted growth, and bone and joint pain. Now that CRYSVITA has received EU approval for self-administration, healthcare professionals can give XLH patients and caregivers more independence. Adult XLH patients and their carers or the parents or legal guardians of children or adolescents suffering from XLH can use the option of self-administration of CRYSVITA. The decision as to whether CRYSVITA can be self-administered is at the discretion of the attending physician and is subject to the prerequisite that the persons concerned are qualified and feel able to self-administer CRYSVITA. ”

Abdul Mullick, President of Kyowa Kirin International, commented: “This approval marks another important milestone in the treatment of people with XLH. At Kyowa Kirin, we are committed to improving the quality of life for patients and their families by providing better ways for healthcare professionals to care for patients. With the EU approval for self-administration of CRYSVITA, we have created another valuable administration option for children, adolescents and adults suffering from XLH. CRYSVITA self-administration is a great example of how Kyowa Kirin addresses the needs of doctors and patients and fulfills our mission to make people smile. ”

CRYSVITA is given as a subcutaneous injection.2 Treatment with CRYSVITA must continue to be started by doctors who have experience in the treatment of patients with bone metabolic disorders. When patients are then given a stable dose, the doctor may recommend that the administration be administered by the patient or caregiver after appropriate training. The first self-administered dose after initiating drug treatment or after changing the dose must be done under the supervision of a healthcare professional.

▼ This medicine is subject to additional monitoring.

About X-linked hypophosphatemia

X-linked hypophosphatemia (XLH) is a rare, genetic condition that causes abnormalities in bones, muscles, and joints.2, 3 XLH disease is not life threatening, but it is lifelong and progressive and can affect the quality of life.4

People with XLH have a genetic defect on the X chromosome that causes increased excretion of phosphate in the urine and poor absorption in the intestine, which leads to chronically low phosphate levels in the blood.4, 5 Phosphate is an important mineral that plays a key role in the energy metabolism of the human body and is required for muscle function and the formation of healthy bones and teeth.6, 7 XLH is not curable, but therapies that rebalance the phosphate levels in the body can help alleviate the symptoms of the disease.8

XLH is the most common form of hereditary rickets.9 It can also occur in people whose families do not have the disease, but it is usually inherited from a parent who is the carrier of the defective gene.10

About CRYSVITA® (Burosumab)

CRYSVITA (burosumab) was developed by Kyowa Kirin and is a recombinant, fully human IgG1 monoclonal antibody against the phosphating hormone fibroblast growth factor 23 (FGF23). FGF23 is a hormone that lowers serum phosphate levels by regulating phosphate excretion and the active production of vitamin D in the kidneys. The loss of phosphate in X-linked hypophosphatemia (XLH) and the resulting drop in the blood phosphate level are caused by an excess of FGF23. CRYSVITA binds to FGF23 and thereby inhibits its biological activity. By blocking excess FGF23, CRYSVITA increases phosphate reabsorption from the kidneys and the production of active vitamin D, which improves the absorption of phosphate and calcium in the intestine.

CRYSVITA has been used clinically since 2018. The preparation was first approved by the European Commission, which granted conditional marketing authorization for CRYSVITA for the treatment of X-linked hypophosphatemia with radiographic evidence of bone disease in children from one year of age and in adolescents with growing skeletons. In 2020, this approval was then also extended to older adolescents and adults.2

CRYSVITA is approved by the US FDA for XLH patients 6 months and older and Health Canada, the Canadian Department of Health, for XLH patients 1 year and older.11, 12

In 2019, CRYSVITA was approved by the Japanese Ministry of Health, Labor and Welfare for the treatment of FGF23-related hypophosphatemic rickets and osteomalacia. In 2020, the cost of CRYSVITA as a self-injecting preparation for the treatment of FGF23-related hypophosphatemic rickets and osteomalacia was covered by the Japanese national health insurance company, NHI.

In January 2020, CRYSVITA was approved by Swissmedic, the Swiss regulatory and supervisory authority for drugs and medical devices, for the treatment of adults, adolescents and children (from the age of one year) suffering from XLH.13

In June 2020, the US FDA approved CRYSVITA for the treatment of patients two years of age and older with tumor-induced osteomalacia (TIO). It is a rare disease characterized by the development of tumors that lead to weakened and soft bones.14

Kyowa Kirin and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE: Ultragenyx) are working together to develop and commercialize CRYSVITA worldwide. The cooperation is based on the cooperation and license agreement between Kyowa Kirin and Ultragenyx.

About Kyowa Kirin

Kyowa Kirin strives to develop and provide novel drugs that improve the quality of life for patients. The global specialty pharmaceutical company based in Japan, which can look back on more than 70 years of tradition, applies the latest scientific findings, including the company’s expertise in antibody research and antibody engineering, to meet the needs of patients and the Society in diverse therapeutic areas such as nephrology, oncology, immunology / allergy and neurology. In the four regions where we do business – Japan, Asia Pacific, North America and EMEA / International – we focus on our goal of making people smile. We are united by our common values: commitment to life, teamwork / wa, innovation and integrity. For more information on Kyowa Kirin’s business, please visit: https://www.kyowakirin.com/

Kyowa Kirin International

http://www.international.kyowa-kirin.com / www.kyowakirin.com

Galabank Business Park

Galashiels, TD1 1QH

United Kingdom

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1 European Medicines Agency. CRYSVITA EPAR product information. Summary of Product Characteristics. Available at: Crysvita, INN-burosumab; (europa.eu). Last update: 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, solution for injection (burosumabum). Available at: https://www.swissmedic.ch/swissmedic/en/home/humanarzneimittel/authorisations/new-medicines/vrysvita-injektionsloesung_burosumabum.html. Last update: January 2020. Last accessed: July 2021.

14 FDA. Verfügbar unter: FDA Approves First Therapy for Rare Disease that Causes Low Phosphate Blood Levels, Bone Softening | FDA. Letzter Zugriff: Juli 2021

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Quelle: Business Wire

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