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Ultragenyx-Kyowa Kirin drug label expanded by US FDA in rare skeletal disorder

Ultragenyx Pharmaceutical Inc. and Tokyo-based Kyowa Kirin Co. Ltd. said their product Crysvita has been approved by the U.S. Food and Drug Administration to treat children as young as six months old with a rare inherited skeletal disorder.

Crysvita, or burosumab, is already approved by the regulatory body to treat X-linked hypophosphatemia, or XLH, in adults and children ages one and older. XLH causes low phosphate levels in the blood, leading to soft, weak bones, which can result in life-long physical disabilities and pain. This leads to an increased risk of fractures in adults and rickets in children, which can lead to lower-body deformity, delays in growth and decreased height.

Infants as young as six months of age can now receive Crysvita. The drug's label was also expanded to include new data, including a phase 3 study in adults that Crysvita improves stiffness and continuously heals fractures.

Other data includes a 64-week-long phase 3 study in children comparing Crysvita with conventional therapy. The standard of care for this disorder is oral phosphate and active vitamin D. The results showed that the drug improved the severity of rickets, lower limb deformity and growth, Kyowa Kirin said in a Sept. 30 press release.

Crysvita received the European Commission's conditional approval in February 2018 to treat XLH in children that are at least 1 year old. The U.K.'s National Institute for Health and Care Excellence also backed the use of the therapy within the country's National Health Service, months after rejecting it in draft guidance issued in June 2018.