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Kyowa Kirin receives recommendation for expanded approval of CRYSVITA®

Mark McIntyre blog

TOKYO, Japan, 24 July 2020 – Kyowa Kirin Co., Ltd. (TSE:4151, Kyowa Kirin) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the expanded approval of CRYSVITA® (burosumab) to include older adolescents and adults living with the rare disease X-linked hypophosphataemia (XLH).

The European Commission has already granted a conditional marketing authorisation for CRYSVITA for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older and adolescents with growing skeletons. The CHMP recommends that this approval is expanded to include all adolescents with radiographic evidence of bone disease, regardless of growth status, as well as adults with XLH. The CHMP opinion will now be reviewed by the European Commission, with a final decision expected in September 2020.

The signs and symptoms of XLH begin in early childhood causing lower limb deformities, shortened stature and pain. These can lead to difficulties with walking and physical functioning, affecting quality of life. The skeletal deformities coupled with unresolved hypophosphataemia mean the disease continues to progress in adults causing pain and stiffness, and multiple musculoskeletal deficits that can affect patients as early as in the second or third decade of life.

“Today’s positive CHMP opinion marks a crucial step forward for the XLH community,” said Abdul Mullick, President of Kyowa Kirin International. “There is currently no approved therapy in Europe for older adolescents and adults with XLH that targets the underlying cause of this debilitating, progressive and life-long disease. Should CRYSVITA be approved for expanded use, it will enable adolescents to continue to receive the benefits of treatment after their bones have stopped growing, and offer adults with XLH a treatment that has been shown to reduce pain and stiffness, improve physical functioning and mobility, and heal pseudofractures and fractures. This expansion of the indication to a wider population aligns perfectly with our vision as a company and our commitment to life that guides our actions every day.”

The positive opinion from the CHMP 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 trial investigating the effects of burosumab on osteomalacia in adults with XLH.

“Adult XLH patients’ response to conventional therapy, which includes phosphate and activated vitamin D, is variable and the evidence-base for its efficacy is limited,” said Dr Karine Briot, Hôpital Cochin, Paris, France. “Having access to an efficacious treatment consistently from childhood through adulthood will be highly valuable to patients and to the physicians administering their care. Today’s recommendation is an important step forward for all people with XLH and those who care for and support them.”

“This achievement forms part of our mission to respond to the requests and hopes of patients living with diseases for which there is currently no adequate treatment,” said Yoshihiro Furuya, SVP, Global Product Lead for CRYSVITA, GPMO of Kyowa Kirin. “We share this milestone with patients, their families and clinical investigators across Europe whose perseverance and commitment have made this progress possible.”

Read the full press release here.