Kyowa Hakko Kirin Co. Ltd, (Kyowa Hakko Kirin), Kyowa Kirin International PLC (Kyowa Kirin International) and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE: Ultragenyx) today announce that Crysvita (burosumab) has received a positive European Commission decision granting a conditional marketing authorisation to Kyowa Kirin for the treatment of X-linked hypophosphatemia (XLH) with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons. XLH is a rare, chronic progressive musculoskeletal disorder that affects children and adults.
This is the first regulatory approval globally for Crysvita, an anti-FGF23 fully human monoclonal antibody that is the first treatment to target the underlying pathophysiology of XLH. The European Medicines Agency recently acknowledged Crysvita as an outstanding contribution to public health and a significant improvement in the endocrinology therapeutic area.
The European Marketing Authorisation is valid in the 28 countries of the European Union and in Norway, Iceland and Liechtenstein. The first commercial launch of Crysvita is expected to take place in Germany in the second quarter of 2018, followed by other European countries.
“Today’s news brings hope to people affected by XLH in Europe, and it’s exciting that Europe is the first global regulatory approval for Crysvita,” said Dr Tom Stratford, President and Chief Executive Officer of Kyowa Kirin International. “We will now focus our efforts on working with health authorities to ensure patient access in European countries. We will also continue our work with the healthcare and patient community to further develop our understanding of the real-world experience of people affected by XLH. This will help to ensure appropriate patient identification and diagnosis and improve standards of care for this rare condition.”
“Throughout our 20-year journey of scientific discovery with XLH and burosumab, we have been guided by the needs of the people affected by this debilitating condition,” said Mitsuo Satoh, Ph.D., Executive Officer, Vice President Head of R&D Division of Kyowa Hakko Kirin. “We look forward to continuing our scientific endeavours in nephrology and bone metabolism to help improve outcomes for these patients.”
“XLH is extremely debilitating to patients and this authorisation in Europe provides children with the first treatment option that addresses the excess FGF23 activity in XLH,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “Through this authorisation and our work throughout the rest of the world we are committed to bringing Crysvita to all patients with XLH who could benefit from the therapy.”
The authorisation follows a positive opinion adopted on 15 December 2017 by the European Committee for Medicinal Products for Human Use to recommend conditional approval of Crysvita for the treatment of X-linked hypophosphatemia (XLH) with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons. The CHMP recommendation is based on data from clinical trials of the antibody for paediatric XLH.
Kyowa Hakko Kirin, Kyowa Kirin International, a wholly owned subsidiary of Kyowa Hakko Kirin, and Ultragenyx have been collaborating in the development and commercialisation of burosumab globally, based on the collaboration and licence agreement between Kyowa Hakko Kirin and Ultragenyx.
About X-Linked Hypophosphatemia (XLH)
XLH is a rare, chronic progressive musculoskeletal disorder characterised by renal phosphate wasting caused by excess FGF23 production, and is inherited as an X-linked dominant trait affecting both males and females. XLH is first seen in infants and also affects adults.
In children, XLH causes skeletal disease, leading to lower-extremity deformity and diminished height.
The conventional treatment of XLH consists of multiple daily doses of phosphate and active vitamin D to counteract the excess effects of FGF23 but does not correct the underlying disease.