Kyowa Kirin Announces European Commission (EC) Approval of CRYSVITA® (burosumab) for the Treatment of X-Linked Hypophosphataemia (XLH) in Older Adolescents and Adults
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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
“Today’s decision from the
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
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
In 2018, the
In 2019, CRYSVITA received approval from
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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:
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.
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®.
9 Lo SH, Lachmann R, Williams A, et al. Exploring the burden of X-linked hypophosphatemia: a European multi-country qualitative study.
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
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
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.
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
19 CRYSVITA Prescribing Information. Available at: https://www.ultragenyx.com/file.cfm/29/docs/Crysvita_Full_Prescribing_Information.pdf. Last updated:
21 Swissmedic. Crysvita, injektionslösung (burosumabum). Available at: https://www.swissmedic.ch/swissmedic/en/home/humanarzneimittel/authorisations/new-medicines/vrysvita-injektionsloesung_burosumabum.html. Last updated:
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