RARE Daily

European Commission Approves Kyowa Kirin’s Crysvita for XLH in Older Adolescents and Adults

October 5, 2020

Rare Daily Staff

The European Commission has approved Kyowa Kirin’s Crysvita for use in older adolescents and adults with the rare disease X-linked hypophosphatemia.

Crysvita was previously approved for the treatment of X-linked hypophosphatemia (XLH) with radiographic evidence of bone disease in children one year of age and older and adolescents with growing skeletons. With this expanded approval, all adolescents with radiographic evidence of bone disease, regardless of growth status, as well as adults with XLH are now also eligible for treatment with Crysvita.

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. Symptoms such as dental abscesses, osteoarthritis, issues with the tendons, and hearing loss may also develop during adulthood. As a result of the disease, some adults may require special equipment to improve their mobility. The physical limitations, as well as pain and stiffness caused by XLH, can affect people’s ability to work and socialize, their emotional wellbeing, and their capacity for self-care.

Crysvita 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 hypophosphatemia in XLH is caused by excessive levels and activity of FGF23. Crysvita is designed to inhibit the biological activity of FGF2 and increase phosphate reabsorption from the kidney, and increase the production of vitamin D, which enhances intestinal absorption of phosphate and calcium.

“Today’s decision from the European Commission is a significant milestone for the management of XLH, a progressive and life-long disease that profoundly impacts the lives of both children and adults,” said Abdul Mullick, president of Kyowa Kirin International. “We now will focus on ensuring access for as many people as possible in this expanded group of eligible patients.”

The application to expand the marketing authorization was based on data from two phase 3 studies that found Crysvita 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.

The safety profile was consistent with that observed in other Crysvita studies, with adverse events including injection site reactions, hyperphosphatemia, and hypersensitivity. There were no treatment-related serious adverse events.


Photo: Abdul Mullick, President of Kyowa Kirin International

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