Ultragenyx and Kyowa Kirin Announce Health Canada Approval of Crysvita™ (burosumab injection) for the Treatment of X–linked Hypophosphatemia (XLH) in Adults and Children
“This approval of Crysvita offers Canadian patients living with XLH the first treatment option that targets the underlying cause of this rare and debilitating disease,” said
“With this approval for Crysvita, now there is an entirely new option for XLH patients in Canada,” said
“Burosumab is the first treatment to address the fundamental problem in this disease – renal phosphate wasting,” said
“Burosumab is a most welcome addition to the treatment options for XLH - a genetically transmitted form of rickets. This treatment has been shown to heal rickets in children and heal fractures and reduce stiffness in adult XLH patients, and is a breakthrough in the treatment of this disabling condition,” says Dr.
Ultragenyx offers a patient assistance program called UltraCare™, to provide ongoing support to patients and their caregivers and to assist them in finding access solutions to Crysvita. The UltraCare team in
About X-Linked Hypophosphatemia (XLH)
XLH is a rare, hereditary, progressive and lifelong skeletal disorder characterized by renal phosphate wasting caused by excess FGF23 production. It affects both children and adults. In children, XLH causes rickets that leads to delayed growth and decreased height. Adults with XLH have an increased risk of fractures, softening of the bones, and stiffness.
Crysvita is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Hakko Kirin, against the phosphaturic hormone fibroblast growth factor 23 (FGF23). FGF23 is a hormone that reduces serum levels of phosphorus and active vitamin D by regulating phosphate excretion and active vitamin D production by the kidney. Phosphate wasting in 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.
For the pediatric XLH population, Health Canada’s approval of Crysvita is supported by 64-week data from Study CL201, a randomized, open-label study in 52 patients ages 5 to 12, which showed that treatment with Crysvita improved rickets, increased serum phosphorus levels, decreased serum alkaline phosphatase activity, and increased growth. The indication is also supported by 40-week data from Study CL205, an open-label study in 13 patients ages 1 to 4. In these patients, Crysvita improved rickets, increased serum phosphorus levels and decreased serum alkaline phosphatase activity.
For the adult XLH indication, Health Canada’s approval of Crysvita is supported by 24-week data from Study CL303, a randomized, double-blind, placebo-controlled study in 134 adult XLH patients. Crysvita treatment resulted in a higher proportion of patients achieving serum phosphorus levels above the lower limit of normal, a higher rate of complete healing of active fractures and pseudofractures, and a decline in the WOMAC stiffness score compared to placebo. The adult indication is also supported by data from the 48-week, open-label, single-arm bone biopsy study in 14 adult XLH patients, which showed healing of osteomalacia as demonstrated by decreases in osteoid volume/bone volume, osteoid thickness and mineralization lag time.
Kyowa Hakko Kirin,
INDICATION AND CLINICAL USE (In Canada)
CRYSVITA (Burosumab Injection) is indicated for the treatment of X-linked hypophosphataemia (XLH) in adult and pediatric patients 1 year of age and older.
Treatment should be initiated and monitored by a health professional experienced in the management of patients with metabolic bone diseases.
Safety and efficacy in geriatric populations have not been established.
- Do not use CRYSVITA with oral phosphate and active vitamin D analogues.
- Do not initiate CRYSVITA treatment if serum phosphorus is within or above the normal range for age.
- CRYSVITA is contraindicated in patients with severe renal impairment or end stage renal disease because these conditions are associated with abnormal mineral metabolism.
RELEVANT WARNINGS AND PRECAUTIONS
- Hyperphosphataemia and risk of ectopic mineralization
- Injection site reactions
- Vitamin D decrease
- Driving and operating machinery
- Hypersensitivity reactions
- Monitoring and laboratory tests
- Pregnancy and breast-feeding
Please find the Product Monograph at http://international.kyowa-kirin.com/ca/crysvita/pm/, which also includes important information relating to adverse reactions and dosing information. The Product Monograph is also available by calling 1-833-388-5872.
Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are no approved therapies.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.
Kyowa Hakko Kirin Co., Ltd. is a research-based life sciences company, with special strengths in biotechnologies. In the core therapeutic areas of oncology, nephrology and immunology/allergy, Kyowa Hakko Kirin leverages leading-edge biotechnologies centred on antibody technologies, to continually discover innovative new drugs and to develop and market those drugs world-wide. In this way, the company is working to realise its vision of becoming a
You can learn more about the business at: www.kyowa-kirin.com.
Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding the availability of Crysvita in
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Source: Ultragenyx Pharmaceutical Inc.