Kyowa Kirin announces EU approval for self-administration of CRYSVITA® ▼ (burosumab) for the treatment of X-linked hypophosphatemia (XLH)

TOKYO–(BUSINESS WIRE) – Kyowa Kirin Co., Ltd. (TSE: 4151, Kyowa Kirin) announced today that CRYSVITA® (burosumab) has been approved in the European Union (EU) as a self-administration option for the treatment of X-linked hypophosphataemia (XLH), a rare metabolic bone disease that affects children and adults Affects adults. The approval means that some patients or caregivers may be suitable, on the recommendation of the treating physician, to self-administer CRYSVITA in its approved indication for the treatment of XLH in children and adolescents aged 1 to 17 years with radiologically proven bone disease, and in adults. 1

Dr. Raja Padidela, Specialist in Pediatric Endocrinology at Royal Manchester Children’s Hospital, UK, said: “XLH is a progressive disabling disease that causes rickets, lower limb deformity, stunted growth, bone and joint pain. Now that CRYSVITA is approved for self-administration in Europe, healthcare professionals have the opportunity to help XLH patients and their caregivers become more independent. Adults with XLH and their caregivers, or the parents or guardians of young children or adolescents with XLH, may benefit from the CRYSVITA self-administration option. Self-administration of CRYSVITA is at the discretion of the treating physician, provided that self-administration is competent and comfortable. ”

Abdul Mullick, President of Kyowa Kirin International, said, “This approval is another important milestone in the treatment of people with XLH. Kyowa Kirin is committed to improving the lives of patients and their families by providing better ways for healthcare professionals to care for them. With the EU approval for self-administration of CRYSVITA, we have created another valuable option in the care of children, adolescents and adults with XLH. CRYSVITA’s self-administration is a great example of how Kyowa Kirin fulfills the needs of doctors and patients and our purpose of making people smile. ”

CRYSVITA is given as a subcutaneous injection.2 Treatment with CRYSVITA must still be started by a doctor who has experience in the management of patients with metabolic bone disorders. If the patient is subsequently given a stable dose, the doctor may recommend that the administration can be carried out by the patient or the nursing staff after appropriate training. The first self-administered dose after starting the drug or changing the dose must be carried out 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 disease that causes abnormalities in bones, muscles, and joints.2,3 XLH is not life threatening, but its burden is lifelong and progressive and can affect a person’s quality of life

People with XLH have a genetic defect on the X chromosome that results in excessive urinary phosphate loss and poor absorption from the intestines, resulting in chronically low levels of phosphate in the blood.4,5 Phosphate is an important mineral that is needed to maintain the body’s energy levels, muscle function, and the formation of healthy bones and teeth.6,7 Although there is no cure for XLH, therapies aimed at restoring phosphate levels in the body can help alleviate symptoms of. to improve the disease.8

XLH is the most common form of hereditary rickets.9 It can sometimes appear in people who have never had the disease in a family history, but is usually passed on from a parent who carries the defective gene.10

About CRYSVITA® (burosumab)

CRYSVITA (burosumab) was developed and developed by Kyowa Kirin and is a recombinant fully human IgG1 monoclonal antibody to the phosphorous hormone fibroblast growth factor 23 (FGF23). FGF23 is a hormone that lowers serum phosphate levels by regulating phosphate excretion and active vitamin D production by the kidney. Phosphate waste and the resulting hypophosphatemia in X-linked hypophosphatemia (XLH) is caused by excess FGF23. CRYSVITA is said to bind to the biological activity of FGF23 and thereby inhibit the biological activity. By blocking excess FGF23 in patients, CRYSVITA is said to increase phosphate reabsorption from the kidney and increase the production of active vitamin D, which improves the intestinal absorption of phosphate and calcium.

CRYSVITA has been available for clinical use since 2018. The first approval was granted by the European Commission, which granted conditional marketing authorization for CRYSVITA for the treatment of XLH with radiological evidence of a bone disease in children from one year of age and adolescents with growing skeletons. In 2020, this authorization was then extended to older adolescents and adults. 2

CRYSVITA is approved by the U.S. Food and Drug Administration (FDA) for patients with XLH 6 months and older and Health Canada for patients with XLH 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, CRYSVITA was reimbursed by National Health Insurance (NHI) of Japan as a self-injecting drug for the treatment of FGF23-related hypophosphatemic rickets and osteomalacia.

In January 2020, Swissmedic launched CRYSVITA for the treatment of adults, adolescents and children (from one year of age) with XLH.13. authorized

In June 2020, the U.S. Food and Drug Administration (FDA) approved CRYSVITA for patients two years of age and older with tumor-induced osteomalacia (TIO), a rare disease characterized by the development of tumors that cause weakened and softened bones. 14

Kyowa Kirin and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE: Ultragenyx) are collaborating to develop and commercialize CRYSVITA worldwide based on the collaboration and licensing agreement between Kyowa Kirin and Ultragenyx.

About Kyowa Kirin

Kyowa Kirin is committed to developing and delivering novel medicines with life changing value. As a Japan-based global specialty pharmaceutical company with more than 70 years of history, the company applies cutting-edge science including an expertise in antibody research and engineering to meet the needs of patients and society in multiple therapeutic areas such as nephrology, oncology, immunology / Allergy and Neurology. In our four regions – Japan, Asia Pacific, North America and EMEA / International – we focus on our goal of making people smile and share our common values ​​of commitment to life, teamwork / wa, innovation and integrity. To learn more about 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

References

1 European Medicines Agency. CRYSVITA EPAR product information. Summary of the product features. Available from: Crysvita, INN-burosumab; (europa.eu). Last updated: June 2021. Last accessed: July 2021.

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

3 D. Haffner, F. Emma, ​​DM Eastwood et al. Consensus Statement. Evidence-based guide. Recommendations from clinical practice for the diagnosis and treatment of X-linked hypophosphatemia. Nat. Rev Nephrol. 2019; 15; 435-455.

4 A. Skrinar, M. Dvorak-Ewell, A. Evins et al. The Lifelong Impact of X-Linked Hypophosphatemia: Results of 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 measurements of osteomalacia in adults with X-linked hypophosphatemia: a single-arm, international phase 3 study. J Bone Miner Res. 2019; 34: 2183-2191.

9 Zimmermann 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 the Promotion of Translation Studies. 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. Executive 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/de/home/humanarzneimittel/authorisations/new-medicines/vrysvita-injektionsloesung_burosumabum.html. Last updated: January 2020. Last accessed: July 2021.

14 FDA. Obtainable from: FDA Approves First Therapy for Rare Diseases That Cause Low Blood Phosphate Levels and Bone Softening | FDA. Last accessed July 2021